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INVESTORS & MEDIA
Investors & Media. Ionis is focused on delivering RNA-targeted therapeutics with transformational potential. Our antisense oligonucleotide drug discovery platform has transformed the lives of patients with devastating diseases, including SMA, hATTR, and FCS. We have the potential to treat many additional diseases where othertherapeutic
ION363 | IONIS PHARMACEUTICALS, INC. ION363. ION363 is an investigational antisense medicine designed to reduce the production of the Fused in Sarcoma (FUS) protein. ION363 is also known as Jacifusen (not an official USAN name) in honor of Jaci Hermstad, the first patient treated with the drug under an expanded access program. It is in development for patients with a rare genetic ION373* | IONIS PHARMACEUTICALS, INC. ION373*. ION373 is an investigational antisense medicine targeting glial fibrillary acidic protein (GFAP) messenger ribonucleic acid (mRNA). ION373 is designed to inhibit the production of GFAP and is being developed as a potential therapy for Alexander disease (AxD). Nearly all cases of AxD are caused by gain-of-function mutations inGFAP that
ION449 | IONIS PHARMACEUTICALS, INC. ION449. Chemistry: Generation 2.5 LICA. ION449 (formerly IONIS-AZ4-2.5-L Rx), also known as AZD8233, is a ligand-conjugated (LICA) investigational antisense medicine designed to reduce plasma levels of proprotein convertase subtilisin/kexin type 9 (PCSK9).PCSK9 is integrally involved in the regulation of LDL-cholesterol. Genetic studies have shown that individuals with life-long reduction of ION541* | IONIS PHARMACEUTICALS, INC. ION541, also known as BIIB105, is an investigational antisense medicine targeting ataxin 2 (ATXN2) RNA. ION541 is designed to reduce the production of ATXN2 protein. It is in development as a potential therapy for patients with most forms of Amyotrophic Lateral Sclerosis (ALS), regardless of family history. Aggregates of TDP-43 protein inmotor
STANLEY T. CROOKE, PH.D., M.D, TO RETIRE FROM IONIS TO CARLSBAD, Calif., Dec. 17, 2020 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), the leader in RNA-targeted therapeutics, announced today that its founder and executive chairman Stanley T. Crooke, M.D., Ph.D., will retire from Ionis and its board of directors effective June 2021. Dr. Crooke is stepping down so that he may focus on his scientific interests and the nonprofit B. LYNNE PARSHALL, ESQ. B. Lynne Parshall, Esq. Senior Strategic Advisor, Ionis. Ms. Parshall has served on the Ionis board since September 2000 and as a senior strategic advisor to Ionis since January 2018. Previously she served as our chief operating officer from December 2007 through December 2017 and as chief financial officer from June 1994 through December2012.
IONIS EARNS $10 MILLION MILESTONE PAYMENT FROM BIOGEN FOR CARLSBAD, Calif., July 17, 2017 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) announced today that it has earned a $10 million milestone payment from Biogen associated with the validation of an undisclosed neurological disease target. Ionis and Biogen have a broad strategic collaboration to develop drugs to treat patients with neurological disorders. IONIS LICENSES NOVEL ANTISENSE DRUG FOR THE TREATMENT OF "CNM is a rare, debilitating disease affecting children and young adults and results in progressive muscular weakness. We are pleased that Dynacure has opted to license IONIS-DNM2-2.5 Rx, which we believe has the potential to bring benefit to patients with CNM," said Brett P. Monia, senior vice president of drug discovery and franchise leader for oncology and rare diseases at Ionis HOME | IONIS PHARMACEUTICALS, INC.ABOUTIONIS INNOVATIONOUR MEDICINESPATIENTS & COMMUNITYINVESTORS & MEDIACAREERS A perpetual and efficient innovation machine. Our drug discovery platform has served as a springboard for drug discovery and realized hope for patients with unmet needs. Our broad, diverse pipeline has more than 40 first-in-class and/or best-in-class medicines designedINVESTORS & MEDIA
Investors & Media. Ionis is focused on delivering RNA-targeted therapeutics with transformational potential. Our antisense oligonucleotide drug discovery platform has transformed the lives of patients with devastating diseases, including SMA, hATTR, and FCS. We have the potential to treat many additional diseases where othertherapeutic
ION363 | IONIS PHARMACEUTICALS, INC. ION363. ION363 is an investigational antisense medicine designed to reduce the production of the Fused in Sarcoma (FUS) protein. ION363 is also known as Jacifusen (not an official USAN name) in honor of Jaci Hermstad, the first patient treated with the drug under an expanded access program. It is in development for patients with a rare genetic ION373* | IONIS PHARMACEUTICALS, INC. ION373*. ION373 is an investigational antisense medicine targeting glial fibrillary acidic protein (GFAP) messenger ribonucleic acid (mRNA). ION373 is designed to inhibit the production of GFAP and is being developed as a potential therapy for Alexander disease (AxD). Nearly all cases of AxD are caused by gain-of-function mutations inGFAP that
ION449 | IONIS PHARMACEUTICALS, INC. ION449. Chemistry: Generation 2.5 LICA. ION449 (formerly IONIS-AZ4-2.5-L Rx), also known as AZD8233, is a ligand-conjugated (LICA) investigational antisense medicine designed to reduce plasma levels of proprotein convertase subtilisin/kexin type 9 (PCSK9).PCSK9 is integrally involved in the regulation of LDL-cholesterol. Genetic studies have shown that individuals with life-long reduction of ION541* | IONIS PHARMACEUTICALS, INC. ION541, also known as BIIB105, is an investigational antisense medicine targeting ataxin 2 (ATXN2) RNA. ION541 is designed to reduce the production of ATXN2 protein. It is in development as a potential therapy for patients with most forms of Amyotrophic Lateral Sclerosis (ALS), regardless of family history. Aggregates of TDP-43 protein inmotor
STANLEY T. CROOKE, PH.D., M.D, TO RETIRE FROM IONIS TO CARLSBAD, Calif., Dec. 17, 2020 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), the leader in RNA-targeted therapeutics, announced today that its founder and executive chairman Stanley T. Crooke, M.D., Ph.D., will retire from Ionis and its board of directors effective June 2021. Dr. Crooke is stepping down so that he may focus on his scientific interests and the nonprofit B. LYNNE PARSHALL, ESQ. B. Lynne Parshall, Esq. Senior Strategic Advisor, Ionis. Ms. Parshall has served on the Ionis board since September 2000 and as a senior strategic advisor to Ionis since January 2018. Previously she served as our chief operating officer from December 2007 through December 2017 and as chief financial officer from June 1994 through December2012.
IONIS EARNS $10 MILLION MILESTONE PAYMENT FROM BIOGEN FOR CARLSBAD, Calif., July 17, 2017 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) announced today that it has earned a $10 million milestone payment from Biogen associated with the validation of an undisclosed neurological disease target. Ionis and Biogen have a broad strategic collaboration to develop drugs to treat patients with neurological disorders. IONIS LICENSES NOVEL ANTISENSE DRUG FOR THE TREATMENT OF "CNM is a rare, debilitating disease affecting children and young adults and results in progressive muscular weakness. We are pleased that Dynacure has opted to license IONIS-DNM2-2.5 Rx, which we believe has the potential to bring benefit to patients with CNM," said Brett P. Monia, senior vice president of drug discovery and franchise leader for oncology and rare diseases at IonisINVESTORS & MEDIA
Investors & Media. Ionis is focused on delivering RNA-targeted therapeutics with transformational potential. Our antisense oligonucleotide drug discovery platform has transformed the lives of patients with devastating diseases, including SMA, hATTR, and FCS. We have the potential to treat many additional diseases where othertherapeutic
ION449 | IONIS PHARMACEUTICALS, INC. ION449. Chemistry: Generation 2.5 LICA. ION449 (formerly IONIS-AZ4-2.5-L Rx), also known as AZD8233, is a ligand-conjugated (LICA) investigational antisense medicine designed to reduce plasma levels of proprotein convertase subtilisin/kexin type 9 (PCSK9).PCSK9 is integrally involved in the regulation of LDL-cholesterol. Genetic studies have shown that individuals with life-long reduction of TOFERSEN | IONIS PHARMACEUTICALS, INC. Tofersen. Chemistry: Generation 2 +. Tofersen (formerly IONIS-SOD1 Rx), also known as BIIB067, is an investigational antisense medicine targeting superoxide dismutase 1 (SOD1), which is the second most common and best understood genetic cause of amyotrophic lateral sclerosis (ALS).Patients with a mutation in the SOD1 gene develop a form of ALS referred to as SOD1-ALS.IONIS-AGT-L
IONIS-AGT-L. Chemistry: Generation 2 + LICA. IONIS-AGT-L Rx is a ligand-conjugated (LICA) investigational antisense medicine designed to reduce the production of angiotensinogen to decrease blood pressure in patients with treatment resistant hypertension (TRH). Despite availability of generic antihypertensive agents, TRH is a major contributor to cardiovascular and renal disease. IONIS-FB-L | IONIS PHARMACEUTICALS, INC. IONIS-FB-L. Chemistry: Generation 2 + LICA. IONIS-FB-L Rx is a ligand-conjugated (LICA) investigational antisense medicine designed to reduce the production of complement factor B (FB). Genetic association studies have shown that overaction of this cascade has been associated with the development of several complement-mediated diseases, including IgA nephropathy (IgAN). STANLEY T. CROOKE, PH.D., M.D, TO RETIRE FROM IONIS TO CARLSBAD, Calif., Dec. 17, 2020 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), the leader in RNA-targeted therapeutics, announced today that its founder and executive chairman Stanley T. Crooke, M.D., Ph.D., will retire from Ionis and its board of directors effective June 2021. Dr. Crooke is stepping down so that he may focus on his scientific interests and the nonprofit ION541* | IONIS PHARMACEUTICALS, INC. ION541, also known as BIIB105, is an investigational antisense medicine targeting ataxin 2 (ATXN2) RNA. ION541 is designed to reduce the production of ATXN2 protein. It is in development as a potential therapy for patients with most forms of Amyotrophic Lateral Sclerosis (ALS), regardless of family history. Aggregates of TDP-43 protein inmotor
PROMISING NEW DATA FOR IONIS' ANTISENSE MEDICINE TARGETING CARLSBAD, Calif., Nov. 13, 2020 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) announced today that new data for ION449, an investigational antisense medicine designed to reduce plasma levels of proprotein convertase subtilisin/kexin type 9, or PCSK9, were presented today at the American Heart Association (AHA) Scientific Sessions. PCSK9 is integrally involved in the ION224 | IONIS PHARMACEUTICALS, INC. ION224 is a ligand-conjugated (LICA) investigational antisense medicine designed to reduce the production of DGAT2, or diacylglycerol acyltransferase 2, to treat patients with NASH, or nonalcoholic steatohepatitis. NASH is a common liver disease characterized by excessive triglycerides in the liver with concurrent inflammation andcellular damage.
IONIS LICENSES NOVEL ANTISENSE DRUG FOR THE TREATMENT OF "CNM is a rare, debilitating disease affecting children and young adults and results in progressive muscular weakness. We are pleased that Dynacure has opted to license IONIS-DNM2-2.5 Rx, which we believe has the potential to bring benefit to patients with CNM," said Brett P. Monia, senior vice president of drug discovery and franchise leader for oncology and rare diseases at Ionis HOME | IONIS PHARMACEUTICALS, INC.ABOUTIONIS INNOVATIONOUR MEDICINESPATIENTS & COMMUNITYINVESTORS & MEDIACAREERS A perpetual and efficient innovation machine. Our drug discovery platform has served as a springboard for drug discovery and realized hope for patients with unmet needs. Our broad, diverse pipeline has more than 40 first-in-class and/or best-in-class medicines designed IONIS TO HOST 2021 VIRTUAL ANNUAL MEETING OF STOCKHOLDERS Ionis to host 2021 virtual annual meeting of stockholders. May 5, 2021 at 4:05 PM EDT. CARLSBAD, Calif., May 5, 2021 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that management will conduct its 2021 virtual Annual Meeting of Stockholders followed by a general corporate update on Wednesday, June 2, 2021.INVESTORS & MEDIA
Investors & Media. Ionis is focused on delivering RNA-targeted therapeutics with transformational potential. Our antisense oligonucleotide drug discovery platform has transformed the lives of patients with devastating diseases, including SMA, hATTR, and FCS. We have the potential to treat many additional diseases where othertherapeutic
STANLEY T. CROOKE, PH.D., M.D, TO RETIRE FROM IONIS TO CARLSBAD, Calif., Dec. 17, 2020 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), the leader in RNA-targeted therapeutics, announced today that its founder and executive chairman Stanley T. Crooke, M.D., Ph.D., will retire from Ionis and its board of directors effective June 2021. Dr. Crooke is stepping down so that he may focus on his scientific interests and the nonprofit TOFERSEN | IONIS PHARMACEUTICALS, INC. Tofersen. Chemistry: Generation 2 +. Tofersen (formerly IONIS-SOD1 Rx), also known as BIIB067, is an investigational antisense medicine targeting superoxide dismutase 1 (SOD1), which is the second most common and best understood genetic cause of amyotrophic lateral sclerosis (ALS).Patients with a mutation in the SOD1 gene develop a form of ALS referred to as SOD1-ALS. ION363 | IONIS PHARMACEUTICALS, INC. ION363. ION363 is an investigational antisense medicine designed to reduce the production of the Fused in Sarcoma (FUS) protein. ION363 is also known as Jacifusen (not an official USAN name) in honor of Jaci Hermstad, the first patient treated with the drug under an expanded access program. It is in development for patients with a rare genetic PROMISING NEW DATA FOR IONIS' ANTISENSE MEDICINE TARGETING CARLSBAD, Calif., Nov. 13, 2020 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) announced today that new data for ION449, an investigational antisense medicine designed to reduce plasma levels of proprotein convertase subtilisin/kexin type 9, or PCSK9, were presented today at the American Heart Association (AHA) Scientific Sessions. PCSK9 is integrally involved in the VUPANORSEN | IONIS PHARMACEUTICALS, INC. Vupanorsen. Chemistry: Generation 2 + LICA. Vupanorsen, formerly known as IONIS-ANGPTL3-L Rx and AKCEA-ANGPTL3-L Rx, is a ligand-conjugated (LICA) investigational antisense medicine designed to reduce angiopoietin-like 3 protein, or ANGPTL3.Human genetic studies have shown that lower levels of ANGPTL3 are associated with lower plasma triglyceride (TG) and LDL-C and protection against certain IONIS REPORTS POSITIVE TOPLINE PHASE 2 STUDY RESULTS OF IONIS-PKK-L Rx was developed using Ionis' advanced ligand-conjugated antisense (LICA) technology. IONIS-PKK-L Rx is one of Ionis' wholly owned medicines to treat rare diseases.. Hereditary angioedema is a rare autosomal dominant disease that results in recurrent, painful attacks of swelling affecting the arms, legs, face, intestinal trackand airway.
B. LYNNE PARSHALL, ESQ. B. Lynne Parshall, Esq. Senior Strategic Advisor, Ionis. Ms. Parshall has served on the Ionis board since September 2000 and as a senior strategic advisor to Ionis since January 2018. Previously she served as our chief operating officer from December 2007 through December 2017 and as chief financial officer from June 1994 through December2012.
HOME | IONIS PHARMACEUTICALS, INC.ABOUTIONIS INNOVATIONOUR MEDICINESPATIENTS & COMMUNITYINVESTORS & MEDIACAREERS A perpetual and efficient innovation machine. Our drug discovery platform has served as a springboard for drug discovery and realized hope for patients with unmet needs. Our broad, diverse pipeline has more than 40 first-in-class and/or best-in-class medicines designed IONIS TO HOST 2021 VIRTUAL ANNUAL MEETING OF STOCKHOLDERS Ionis to host 2021 virtual annual meeting of stockholders. May 5, 2021 at 4:05 PM EDT. CARLSBAD, Calif., May 5, 2021 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that management will conduct its 2021 virtual Annual Meeting of Stockholders followed by a general corporate update on Wednesday, June 2, 2021.INVESTORS & MEDIA
Investors & Media. Ionis is focused on delivering RNA-targeted therapeutics with transformational potential. Our antisense oligonucleotide drug discovery platform has transformed the lives of patients with devastating diseases, including SMA, hATTR, and FCS. We have the potential to treat many additional diseases where othertherapeutic
STANLEY T. CROOKE, PH.D., M.D, TO RETIRE FROM IONIS TO CARLSBAD, Calif., Dec. 17, 2020 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), the leader in RNA-targeted therapeutics, announced today that its founder and executive chairman Stanley T. Crooke, M.D., Ph.D., will retire from Ionis and its board of directors effective June 2021. Dr. Crooke is stepping down so that he may focus on his scientific interests and the nonprofit TOFERSEN | IONIS PHARMACEUTICALS, INC. Tofersen. Chemistry: Generation 2 +. Tofersen (formerly IONIS-SOD1 Rx), also known as BIIB067, is an investigational antisense medicine targeting superoxide dismutase 1 (SOD1), which is the second most common and best understood genetic cause of amyotrophic lateral sclerosis (ALS).Patients with a mutation in the SOD1 gene develop a form of ALS referred to as SOD1-ALS. ION363 | IONIS PHARMACEUTICALS, INC. ION363. ION363 is an investigational antisense medicine designed to reduce the production of the Fused in Sarcoma (FUS) protein. ION363 is also known as Jacifusen (not an official USAN name) in honor of Jaci Hermstad, the first patient treated with the drug under an expanded access program. It is in development for patients with a rare genetic PROMISING NEW DATA FOR IONIS' ANTISENSE MEDICINE TARGETING CARLSBAD, Calif., Nov. 13, 2020 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) announced today that new data for ION449, an investigational antisense medicine designed to reduce plasma levels of proprotein convertase subtilisin/kexin type 9, or PCSK9, were presented today at the American Heart Association (AHA) Scientific Sessions. PCSK9 is integrally involved in the VUPANORSEN | IONIS PHARMACEUTICALS, INC. Vupanorsen. Chemistry: Generation 2 + LICA. Vupanorsen, formerly known as IONIS-ANGPTL3-L Rx and AKCEA-ANGPTL3-L Rx, is a ligand-conjugated (LICA) investigational antisense medicine designed to reduce angiopoietin-like 3 protein, or ANGPTL3.Human genetic studies have shown that lower levels of ANGPTL3 are associated with lower plasma triglyceride (TG) and LDL-C and protection against certain IONIS REPORTS POSITIVE TOPLINE PHASE 2 STUDY RESULTS OF IONIS-PKK-L Rx was developed using Ionis' advanced ligand-conjugated antisense (LICA) technology. IONIS-PKK-L Rx is one of Ionis' wholly owned medicines to treat rare diseases.. Hereditary angioedema is a rare autosomal dominant disease that results in recurrent, painful attacks of swelling affecting the arms, legs, face, intestinal trackand airway.
B. LYNNE PARSHALL, ESQ. B. Lynne Parshall, Esq. Senior Strategic Advisor, Ionis. Ms. Parshall has served on the Ionis board since September 2000 and as a senior strategic advisor to Ionis since January 2018. Previously she served as our chief operating officer from December 2007 through December 2017 and as chief financial officer from June 1994 through December2012.
OUR MEDICINES
A perpetual and efficient innovation machine. Our platform technology has served as a springboard for drug discovery and realized hope for patients with unmet needs. Our broad, diverse pipeline has more than 40 first-in-class and/or best-in-class medicines designed TOFERSEN | IONIS PHARMACEUTICALS, INC. Tofersen. Chemistry: Generation 2 +. Tofersen (formerly IONIS-SOD1 Rx), also known as BIIB067, is an investigational antisense medicine targeting superoxide dismutase 1 (SOD1), which is the second most common and best understood genetic cause of amyotrophic lateral sclerosis (ALS).Patients with a mutation in the SOD1 gene develop a form of ALS referred to as SOD1-ALS. PROMISING NEW DATA FOR IONIS' ANTISENSE MEDICINE TARGETING CARLSBAD, Calif., Nov. 13, 2020 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) announced today that new data for ION449, an investigational antisense medicine designed to reduce plasma levels of proprotein convertase subtilisin/kexin type 9, or PCSK9, were presented today at the American Heart Association (AHA) Scientific Sessions. PCSK9 is integrally involved in the ION363 | IONIS PHARMACEUTICALS, INC. ION363. ION363 is an investigational antisense medicine designed to reduce the production of the Fused in Sarcoma (FUS) protein. ION363 is also known as Jacifusen (not an official USAN name) in honor of Jaci Hermstad, the first patient treated with the drug under an expanded access program. It is in development for patients with a rare genetic PIPELINE PRECLINICAL Our drug discovery platform has served as a springboard for drug discovery and realized hope for patients with unmet needs. Our broad, diverse pipeline has more than 40 first-in-class and/or best-in-class medicines designed to treat a broad range of diseases including cancer ION373* | IONIS PHARMACEUTICALS, INC. ION373*. ION373 is an investigational antisense medicine targeting glial fibrillary acidic protein (GFAP) messenger ribonucleic acid (mRNA). ION373 is designed to inhibit the production of GFAP and is being developed as a potential therapy for Alexander disease (AxD). Nearly all cases of AxD are caused by gain-of-function mutations inGFAP that
OPTIMIZING ANTISENSE LICA, or Ligand Conjugated Antisense, is a chemical technology we developed at Ionis that involves the attachment of a molecule called a ligand that binds with receptors on the surfaces of cells in a highly specific manner. Because these receptors are often found only on certain cell types, LICA allows us to effectively deliver ourantisense
ION541* | IONIS PHARMACEUTICALS, INC. ION541, also known as BIIB105, is an investigational antisense medicine targeting ataxin 2 (ATXN2) RNA. ION541 is designed to reduce the production of ATXN2 protein. It is in development as a potential therapy for patients with most forms of Amyotrophic Lateral Sclerosis (ALS), regardless of family history. Aggregates of TDP-43 protein inmotor
COMMUNITY ENGAGEMENT Corporate Alliances. Featured. 8/2. Surf Away SMA with Ricochet. La Jolla Shores Beach – Tower 33 La Jolla, CA 92037. In collaboration with Cure SMA, Ionis will continue to support local families effected by SMA for a wonderful event at the beach including food, fun, and surfing. With a limitless pursuit to support patients and families IONIS EARNS $10 MILLION MILESTONE PAYMENT FROM BIOGEN FOR CARLSBAD, Calif., July 17, 2017 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) announced today that it has earned a $10 million milestone payment from Biogen associated with the validation of an undisclosed neurological disease target. Ionis and Biogen have a broad strategic collaboration to develop drugs to treat patients with neurological disorders. HOME | IONIS PHARMACEUTICALS, INC.ABOUTIONIS INNOVATIONOUR MEDICINESPATIENTS & COMMUNITYINVESTORS & MEDIACAREERS A perpetual and efficient innovation machine. Our drug discovery platform has served as a springboard for drug discovery and realized hope for patients with unmet needs. Our broad, diverse pipeline has more than 40 first-in-class and/or best-in-class medicines designed TOFERSEN | IONIS PHARMACEUTICALS, INC. Tofersen. Chemistry: Generation 2 +. Tofersen (formerly IONIS-SOD1 Rx), also known as BIIB067, is an investigational antisense medicine targeting superoxide dismutase 1 (SOD1), which is the second most common and best understood genetic cause of amyotrophic lateral sclerosis (ALS).Patients with a mutation in the SOD1 gene develop a form of ALS referred to as SOD1-ALS. ION363 | IONIS PHARMACEUTICALS, INC. ION363. ION363 is an investigational antisense medicine designed to reduce the production of the Fused in Sarcoma (FUS) protein. ION363 is also known as Jacifusen (not an official USAN name) in honor of Jaci Hermstad, the first patient treated with the drug under an expanded access program. It is in development for patients with a rare genetic PROMISING NEW DATA FOR IONIS' ANTISENSE MEDICINE TARGETING CARLSBAD, Calif., Nov. 13, 2020 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) announced today that new data for ION449, an investigational antisense medicine designed to reduce plasma levels of proprotein convertase subtilisin/kexin type 9, or PCSK9, were presented today at the American Heart Association (AHA) Scientific Sessions. PCSK9 is integrally involved in the ION449 | IONIS PHARMACEUTICALS, INC. ION449. Chemistry: Generation 2.5 LICA. ION449 (formerly IONIS-AZ4-2.5-L Rx), also known as AZD8233, is a ligand-conjugated (LICA) investigational antisense medicine designed to reduce plasma levels of proprotein convertase subtilisin/kexin type 9 (PCSK9).PCSK9 is integrally involved in the regulation of LDL-cholesterol. Genetic studies have shown that individuals with life-long reduction of ION373* | IONIS PHARMACEUTICALS, INC. ION373*. ION373 is an investigational antisense medicine targeting glial fibrillary acidic protein (GFAP) messenger ribonucleic acid (mRNA). ION373 is designed to inhibit the production of GFAP and is being developed as a potential therapy for Alexander disease (AxD). Nearly all cases of AxD are caused by gain-of-function mutations inGFAP that
STANLEY T. CROOKE, PH.D., M.D, TO RETIRE FROM IONIS TO CARLSBAD, Calif., Dec. 17, 2020 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), the leader in RNA-targeted therapeutics, announced today that its founder and executive chairman Stanley T. Crooke, M.D., Ph.D., will retire from Ionis and its board of directors effective June 2021. Dr. Crooke is stepping down so that he may focus on his scientific interests and the nonprofit VUPANORSEN | IONIS PHARMACEUTICALS, INC. Vupanorsen. Chemistry: Generation 2 + LICA. Vupanorsen, formerly known as IONIS-ANGPTL3-L Rx and AKCEA-ANGPTL3-L Rx, is a ligand-conjugated (LICA) investigational antisense medicine designed to reduce angiopoietin-like 3 protein, or ANGPTL3.Human genetic studies have shown that lower levels of ANGPTL3 are associated with lower plasma triglyceride (TG) and LDL-C and protection against certain ION537 WILL BE EVALUATED AT MD ANDERSON IN A PHASE 1 STUDY The Investor Relations website contains information about Ionis Pharmaceuticals, Inc.'s business for stockholders, potential investors, and financial analysts. IONIS EARNS $10 MILLION MILESTONE PAYMENT FROM BIOGEN FOR CARLSBAD, Calif., July 17, 2017 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) announced today that it has earned a $10 million milestone payment from Biogen associated with the validation of an undisclosed neurological disease target. Ionis and Biogen have a broad strategic collaboration to develop drugs to treat patients with neurological disorders. HOME | IONIS PHARMACEUTICALS, INC.ABOUTIONIS INNOVATIONOUR MEDICINESPATIENTS & COMMUNITYINVESTORS & MEDIACAREERS A perpetual and efficient innovation machine. Our drug discovery platform has served as a springboard for drug discovery and realized hope for patients with unmet needs. Our broad, diverse pipeline has more than 40 first-in-class and/or best-in-class medicines designed TOFERSEN | IONIS PHARMACEUTICALS, INC. Tofersen. Chemistry: Generation 2 +. Tofersen (formerly IONIS-SOD1 Rx), also known as BIIB067, is an investigational antisense medicine targeting superoxide dismutase 1 (SOD1), which is the second most common and best understood genetic cause of amyotrophic lateral sclerosis (ALS).Patients with a mutation in the SOD1 gene develop a form of ALS referred to as SOD1-ALS. ION363 | IONIS PHARMACEUTICALS, INC. ION363. ION363 is an investigational antisense medicine designed to reduce the production of the Fused in Sarcoma (FUS) protein. ION363 is also known as Jacifusen (not an official USAN name) in honor of Jaci Hermstad, the first patient treated with the drug under an expanded access program. It is in development for patients with a rare genetic PROMISING NEW DATA FOR IONIS' ANTISENSE MEDICINE TARGETING CARLSBAD, Calif., Nov. 13, 2020 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) announced today that new data for ION449, an investigational antisense medicine designed to reduce plasma levels of proprotein convertase subtilisin/kexin type 9, or PCSK9, were presented today at the American Heart Association (AHA) Scientific Sessions. PCSK9 is integrally involved in the ION449 | IONIS PHARMACEUTICALS, INC. ION449. Chemistry: Generation 2.5 LICA. ION449 (formerly IONIS-AZ4-2.5-L Rx), also known as AZD8233, is a ligand-conjugated (LICA) investigational antisense medicine designed to reduce plasma levels of proprotein convertase subtilisin/kexin type 9 (PCSK9).PCSK9 is integrally involved in the regulation of LDL-cholesterol. Genetic studies have shown that individuals with life-long reduction of ION373* | IONIS PHARMACEUTICALS, INC. ION373*. ION373 is an investigational antisense medicine targeting glial fibrillary acidic protein (GFAP) messenger ribonucleic acid (mRNA). ION373 is designed to inhibit the production of GFAP and is being developed as a potential therapy for Alexander disease (AxD). Nearly all cases of AxD are caused by gain-of-function mutations inGFAP that
STANLEY T. CROOKE, PH.D., M.D, TO RETIRE FROM IONIS TO CARLSBAD, Calif., Dec. 17, 2020 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), the leader in RNA-targeted therapeutics, announced today that its founder and executive chairman Stanley T. Crooke, M.D., Ph.D., will retire from Ionis and its board of directors effective June 2021. Dr. Crooke is stepping down so that he may focus on his scientific interests and the nonprofit VUPANORSEN | IONIS PHARMACEUTICALS, INC. Vupanorsen. Chemistry: Generation 2 + LICA. Vupanorsen, formerly known as IONIS-ANGPTL3-L Rx and AKCEA-ANGPTL3-L Rx, is a ligand-conjugated (LICA) investigational antisense medicine designed to reduce angiopoietin-like 3 protein, or ANGPTL3.Human genetic studies have shown that lower levels of ANGPTL3 are associated with lower plasma triglyceride (TG) and LDL-C and protection against certain ION537 WILL BE EVALUATED AT MD ANDERSON IN A PHASE 1 STUDY The Investor Relations website contains information about Ionis Pharmaceuticals, Inc.'s business for stockholders, potential investors, and financial analysts. IONIS EARNS $10 MILLION MILESTONE PAYMENT FROM BIOGEN FOR CARLSBAD, Calif., July 17, 2017 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) announced today that it has earned a $10 million milestone payment from Biogen associated with the validation of an undisclosed neurological disease target. Ionis and Biogen have a broad strategic collaboration to develop drugs to treat patients with neurological disorders. TOFERSEN | IONIS PHARMACEUTICALS, INC. Tofersen. Chemistry: Generation 2 +. Tofersen (formerly IONIS-SOD1 Rx), also known as BIIB067, is an investigational antisense medicine targeting superoxide dismutase 1 (SOD1), which is the second most common and best understood genetic cause of amyotrophic lateral sclerosis (ALS).Patients with a mutation in the SOD1 gene develop a form of ALS referred to as SOD1-ALS. ION449 | IONIS PHARMACEUTICALS, INC. ION449. Chemistry: Generation 2.5 LICA. ION449 (formerly IONIS-AZ4-2.5-L Rx), also known as AZD8233, is a ligand-conjugated (LICA) investigational antisense medicine designed to reduce plasma levels of proprotein convertase subtilisin/kexin type 9 (PCSK9).PCSK9 is integrally involved in the regulation of LDL-cholesterol. Genetic studies have shown that individuals with life-long reduction ofINVESTORS & MEDIA
Investors & Media. Ionis is focused on delivering RNA-targeted therapeutics with transformational potential. Our antisense oligonucleotide drug discovery platform has transformed the lives of patients with devastating diseases, including SMA, hATTR, and FCS. We have the potential to treat many additional diseases where othertherapeutic
IONIS TO HOST 2021 VIRTUAL ANNUAL MEETING OF STOCKHOLDERS Ionis to host 2021 virtual annual meeting of stockholders. May 5, 2021 at 4:05 PM EDT. CARLSBAD, Calif., May 5, 2021 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that management will conduct its 2021 virtual Annual Meeting of Stockholders followed by a general corporate update on Wednesday, June 2, 2021. ION541* | IONIS PHARMACEUTICALS, INC. ION541, also known as BIIB105, is an investigational antisense medicine targeting ataxin 2 (ATXN2) RNA. ION541 is designed to reduce the production of ATXN2 protein. It is in development as a potential therapy for patients with most forms of Amyotrophic Lateral Sclerosis (ALS), regardless of family history. Aggregates of TDP-43 protein inmotor
OPTIMIZING ANTISENSE LICA, or Ligand Conjugated Antisense, is a chemical technology we developed at Ionis that involves the attachment of a molecule called a ligand that binds with receptors on the surfaces of cells in a highly specific manner. Because these receptors are often found only on certain cell types, LICA allows us to effectively deliver ourantisense
IONIS-PKK-L
IONIS-PKK-L. Chemistry: Generation 2 + LICA. IONIS-PKK-L Rx is a ligand-conjugated (LICA) investigational antisense medicine designed to reduce the production of prekallikrein, or PKK, to treat patients with hereditary angioedema, or HAE. PKK plays an important role in the activation of inflammatory mediators associated with acute attacks ofHAE.
STANLEY T. CROOKE, PH.D., M.D, TO RETIRE FROM IONIS TO CARLSBAD, Calif., Dec. 17, 2020 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), the leader in RNA-targeted therapeutics, announced today that its founder and executive chairman Stanley T. Crooke, M.D., Ph.D., will retire from Ionis and its board of directors effective June 2021. Dr. Crooke is stepping down so that he may focus on his scientific interests and the nonprofit COMMUNITY ENGAGEMENT Corporate Alliances. Featured. 8/2. Surf Away SMA with Ricochet. La Jolla Shores Beach – Tower 33 La Jolla, CA 92037. In collaboration with Cure SMA, Ionis will continue to support local families effected by SMA for a wonderful event at the beach including food, fun, and surfing. With a limitless pursuit to support patients and families IONIS LICENSES NOVEL ANTISENSE DRUG FOR THE TREATMENT OF "CNM is a rare, debilitating disease affecting children and young adults and results in progressive muscular weakness. We are pleased that Dynacure has opted to license IONIS-DNM2-2.5 Rx, which we believe has the potential to bring benefit to patients with CNM," said Brett P. Monia, senior vice president of drug discovery and franchise leader for oncology and rare diseases at Ionis HOME | IONIS PHARMACEUTICALS, INC.ABOUTIONIS INNOVATIONOUR MEDICINESPATIENTS & COMMUNITYINVESTORS & MEDIACAREERS A perpetual and efficient innovation machine. Our drug discovery platform has served as a springboard for drug discovery and realized hope for patients with unmet needs. Our broad, diverse pipeline has more than 40 first-in-class and/or best-in-class medicines designed TOFERSEN | IONIS PHARMACEUTICALS, INC. Tofersen. Chemistry: Generation 2 +. Tofersen (formerly IONIS-SOD1 Rx), also known as BIIB067, is an investigational antisense medicine targeting superoxide dismutase 1 (SOD1), which is the second most common and best understood genetic cause of amyotrophic lateral sclerosis (ALS).Patients with a mutation in the SOD1 gene develop a form of ALS referred to as SOD1-ALS. ION363 | IONIS PHARMACEUTICALS, INC. ION363. ION363 is an investigational antisense medicine designed to reduce the production of the Fused in Sarcoma (FUS) protein. ION363 is also known as Jacifusen (not an official USAN name) in honor of Jaci Hermstad, the first patient treated with the drug under an expanded access program. It is in development for patients with a rare genetic PROMISING NEW DATA FOR IONIS' ANTISENSE MEDICINE TARGETING CARLSBAD, Calif., Nov. 13, 2020 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) announced today that new data for ION449, an investigational antisense medicine designed to reduce plasma levels of proprotein convertase subtilisin/kexin type 9, or PCSK9, were presented today at the American Heart Association (AHA) Scientific Sessions. PCSK9 is integrally involved in the ION449 | IONIS PHARMACEUTICALS, INC. ION449. Chemistry: Generation 2.5 LICA. ION449 (formerly IONIS-AZ4-2.5-L Rx), also known as AZD8233, is a ligand-conjugated (LICA) investigational antisense medicine designed to reduce plasma levels of proprotein convertase subtilisin/kexin type 9 (PCSK9).PCSK9 is integrally involved in the regulation of LDL-cholesterol. Genetic studies have shown that individuals with life-long reduction of ION373* | IONIS PHARMACEUTICALS, INC. ION373*. ION373 is an investigational antisense medicine targeting glial fibrillary acidic protein (GFAP) messenger ribonucleic acid (mRNA). ION373 is designed to inhibit the production of GFAP and is being developed as a potential therapy for Alexander disease (AxD). Nearly all cases of AxD are caused by gain-of-function mutations inGFAP that
STANLEY T. CROOKE, PH.D., M.D, TO RETIRE FROM IONIS TO CARLSBAD, Calif., Dec. 17, 2020 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), the leader in RNA-targeted therapeutics, announced today that its founder and executive chairman Stanley T. Crooke, M.D., Ph.D., will retire from Ionis and its board of directors effective June 2021. Dr. Crooke is stepping down so that he may focus on his scientific interests and the nonprofit VUPANORSEN | IONIS PHARMACEUTICALS, INC. Vupanorsen. Chemistry: Generation 2 + LICA. Vupanorsen, formerly known as IONIS-ANGPTL3-L Rx and AKCEA-ANGPTL3-L Rx, is a ligand-conjugated (LICA) investigational antisense medicine designed to reduce angiopoietin-like 3 protein, or ANGPTL3.Human genetic studies have shown that lower levels of ANGPTL3 are associated with lower plasma triglyceride (TG) and LDL-C and protection against certain ION537 WILL BE EVALUATED AT MD ANDERSON IN A PHASE 1 STUDY The Investor Relations website contains information about Ionis Pharmaceuticals, Inc.'s business for stockholders, potential investors, and financial analysts. IONIS EARNS $10 MILLION MILESTONE PAYMENT FROM BIOGEN FOR CARLSBAD, Calif., July 17, 2017 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) announced today that it has earned a $10 million milestone payment from Biogen associated with the validation of an undisclosed neurological disease target. Ionis and Biogen have a broad strategic collaboration to develop drugs to treat patients with neurological disorders. HOME | IONIS PHARMACEUTICALS, INC.ABOUTIONIS INNOVATIONOUR MEDICINESPATIENTS & COMMUNITYINVESTORS & MEDIACAREERS A perpetual and efficient innovation machine. Our drug discovery platform has served as a springboard for drug discovery and realized hope for patients with unmet needs. Our broad, diverse pipeline has more than 40 first-in-class and/or best-in-class medicines designed TOFERSEN | IONIS PHARMACEUTICALS, INC. Tofersen. Chemistry: Generation 2 +. Tofersen (formerly IONIS-SOD1 Rx), also known as BIIB067, is an investigational antisense medicine targeting superoxide dismutase 1 (SOD1), which is the second most common and best understood genetic cause of amyotrophic lateral sclerosis (ALS).Patients with a mutation in the SOD1 gene develop a form of ALS referred to as SOD1-ALS. ION363 | IONIS PHARMACEUTICALS, INC. ION363. ION363 is an investigational antisense medicine designed to reduce the production of the Fused in Sarcoma (FUS) protein. ION363 is also known as Jacifusen (not an official USAN name) in honor of Jaci Hermstad, the first patient treated with the drug under an expanded access program. It is in development for patients with a rare genetic PROMISING NEW DATA FOR IONIS' ANTISENSE MEDICINE TARGETING CARLSBAD, Calif., Nov. 13, 2020 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) announced today that new data for ION449, an investigational antisense medicine designed to reduce plasma levels of proprotein convertase subtilisin/kexin type 9, or PCSK9, were presented today at the American Heart Association (AHA) Scientific Sessions. PCSK9 is integrally involved in the ION449 | IONIS PHARMACEUTICALS, INC. ION449. Chemistry: Generation 2.5 LICA. ION449 (formerly IONIS-AZ4-2.5-L Rx), also known as AZD8233, is a ligand-conjugated (LICA) investigational antisense medicine designed to reduce plasma levels of proprotein convertase subtilisin/kexin type 9 (PCSK9).PCSK9 is integrally involved in the regulation of LDL-cholesterol. Genetic studies have shown that individuals with life-long reduction of ION373* | IONIS PHARMACEUTICALS, INC. ION373*. ION373 is an investigational antisense medicine targeting glial fibrillary acidic protein (GFAP) messenger ribonucleic acid (mRNA). ION373 is designed to inhibit the production of GFAP and is being developed as a potential therapy for Alexander disease (AxD). Nearly all cases of AxD are caused by gain-of-function mutations inGFAP that
STANLEY T. CROOKE, PH.D., M.D, TO RETIRE FROM IONIS TO CARLSBAD, Calif., Dec. 17, 2020 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), the leader in RNA-targeted therapeutics, announced today that its founder and executive chairman Stanley T. Crooke, M.D., Ph.D., will retire from Ionis and its board of directors effective June 2021. Dr. Crooke is stepping down so that he may focus on his scientific interests and the nonprofit VUPANORSEN | IONIS PHARMACEUTICALS, INC. Vupanorsen. Chemistry: Generation 2 + LICA. Vupanorsen, formerly known as IONIS-ANGPTL3-L Rx and AKCEA-ANGPTL3-L Rx, is a ligand-conjugated (LICA) investigational antisense medicine designed to reduce angiopoietin-like 3 protein, or ANGPTL3.Human genetic studies have shown that lower levels of ANGPTL3 are associated with lower plasma triglyceride (TG) and LDL-C and protection against certain ION537 WILL BE EVALUATED AT MD ANDERSON IN A PHASE 1 STUDY The Investor Relations website contains information about Ionis Pharmaceuticals, Inc.'s business for stockholders, potential investors, and financial analysts. IONIS EARNS $10 MILLION MILESTONE PAYMENT FROM BIOGEN FOR CARLSBAD, Calif., July 17, 2017 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) announced today that it has earned a $10 million milestone payment from Biogen associated with the validation of an undisclosed neurological disease target. Ionis and Biogen have a broad strategic collaboration to develop drugs to treat patients with neurological disorders. TOFERSEN | IONIS PHARMACEUTICALS, INC. Tofersen. Chemistry: Generation 2 +. Tofersen (formerly IONIS-SOD1 Rx), also known as BIIB067, is an investigational antisense medicine targeting superoxide dismutase 1 (SOD1), which is the second most common and best understood genetic cause of amyotrophic lateral sclerosis (ALS).Patients with a mutation in the SOD1 gene develop a form of ALS referred to as SOD1-ALS. ION449 | IONIS PHARMACEUTICALS, INC. ION449. Chemistry: Generation 2.5 LICA. ION449 (formerly IONIS-AZ4-2.5-L Rx), also known as AZD8233, is a ligand-conjugated (LICA) investigational antisense medicine designed to reduce plasma levels of proprotein convertase subtilisin/kexin type 9 (PCSK9).PCSK9 is integrally involved in the regulation of LDL-cholesterol. Genetic studies have shown that individuals with life-long reduction ofINVESTORS & MEDIA
Investors & Media. Ionis is focused on delivering RNA-targeted therapeutics with transformational potential. Our antisense oligonucleotide drug discovery platform has transformed the lives of patients with devastating diseases, including SMA, hATTR, and FCS. We have the potential to treat many additional diseases where othertherapeutic
IONIS TO HOST 2021 VIRTUAL ANNUAL MEETING OF STOCKHOLDERS Ionis to host 2021 virtual annual meeting of stockholders. May 5, 2021 at 4:05 PM EDT. CARLSBAD, Calif., May 5, 2021 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that management will conduct its 2021 virtual Annual Meeting of Stockholders followed by a general corporate update on Wednesday, June 2, 2021. ION541* | IONIS PHARMACEUTICALS, INC. ION541, also known as BIIB105, is an investigational antisense medicine targeting ataxin 2 (ATXN2) RNA. ION541 is designed to reduce the production of ATXN2 protein. It is in development as a potential therapy for patients with most forms of Amyotrophic Lateral Sclerosis (ALS), regardless of family history. Aggregates of TDP-43 protein inmotor
OPTIMIZING ANTISENSE LICA, or Ligand Conjugated Antisense, is a chemical technology we developed at Ionis that involves the attachment of a molecule called a ligand that binds with receptors on the surfaces of cells in a highly specific manner. Because these receptors are often found only on certain cell types, LICA allows us to effectively deliver ourantisense
IONIS-PKK-L
IONIS-PKK-L. Chemistry: Generation 2 + LICA. IONIS-PKK-L Rx is a ligand-conjugated (LICA) investigational antisense medicine designed to reduce the production of prekallikrein, or PKK, to treat patients with hereditary angioedema, or HAE. PKK plays an important role in the activation of inflammatory mediators associated with acute attacks ofHAE.
STANLEY T. CROOKE, PH.D., M.D, TO RETIRE FROM IONIS TO CARLSBAD, Calif., Dec. 17, 2020 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), the leader in RNA-targeted therapeutics, announced today that its founder and executive chairman Stanley T. Crooke, M.D., Ph.D., will retire from Ionis and its board of directors effective June 2021. Dr. Crooke is stepping down so that he may focus on his scientific interests and the nonprofit COMMUNITY ENGAGEMENT Corporate Alliances. Featured. 8/2. Surf Away SMA with Ricochet. La Jolla Shores Beach – Tower 33 La Jolla, CA 92037. In collaboration with Cure SMA, Ionis will continue to support local families effected by SMA for a wonderful event at the beach including food, fun, and surfing. With a limitless pursuit to support patients and families IONIS LICENSES NOVEL ANTISENSE DRUG FOR THE TREATMENT OF "CNM is a rare, debilitating disease affecting children and young adults and results in progressive muscular weakness. We are pleased that Dynacure has opted to license IONIS-DNM2-2.5 Rx, which we believe has the potential to bring benefit to patients with CNM," said Brett P. Monia, senior vice president of drug discovery and franchise leader for oncology and rare diseases at Ionis HOME | IONIS PHARMACEUTICALS, INC.ABOUTIONIS INNOVATIONOUR MEDICINESPATIENTS & COMMUNITYINVESTORS & MEDIACAREERS A perpetual and efficient innovation machine. Our drug discovery platform has served as a springboard for drug discovery and realized hope for patients with unmet needs. Our broad, diverse pipeline has more than 40 first-in-class and/or best-in-class medicines designed CAREERS | IONIS PHARMACEUTICALS, INC.INVESTOR FAQSCONTACTCOMMITTEE COMPOSITIONANTISENSE TECHNOLOGYOUR MEDICINES Ionis is where passionate, driven people come together to help patients in need. Our success is a direct result of our outstanding employees. Located in the North San Diego County city of Carlsbad, we’re an industry leader in the discovery and development of antisense drugs. Our work environment is challenging, motivating,rewarding, and
INVESTORS & MEDIA
Investors & Media. Ionis is focused on delivering RNA-targeted therapeutics with transformational potential. Our antisense oligonucleotide drug discovery platform has transformed the lives of patients with devastating diseases, including SMA, hATTR, and FCS. We have the potential to treat many additional diseases where othertherapeutic
ION373* | IONIS PHARMACEUTICALS, INC. ION373*. ION373 is an investigational antisense medicine targeting glial fibrillary acidic protein (GFAP) messenger ribonucleic acid (mRNA). ION373 is designed to inhibit the production of GFAP and is being developed as a potential therapy for Alexander disease (AxD). Nearly all cases of AxD are caused by gain-of-function mutations inGFAP that
OPTIMIZING ANTISENSE LICA, or Ligand Conjugated Antisense, is a chemical technology we developed at Ionis that involves the attachment of a molecule called a ligand that binds with receptors on the surfaces of cells in a highly specific manner. Because these receptors are often found only on certain cell types, LICA allows us to effectively deliver ourantisense
COMMUNITY ENGAGEMENT Corporate Alliances. Featured. 8/2. Surf Away SMA with Ricochet. La Jolla Shores Beach – Tower 33 La Jolla, CA 92037. In collaboration with Cure SMA, Ionis will continue to support local families effected by SMA for a wonderful event at the beach including food, fun, and surfing. With a limitless pursuit to support patients and familiesANNUAL REPORTS
The Investor Relations website contains information about Ionis Pharmaceuticals, Inc.'s business for stockholders, potential investors, and financial analysts. B. LYNNE PARSHALL, ESQ. B. Lynne Parshall, Esq. Senior Strategic Advisor, Ionis. Ms. Parshall has served on the Ionis board since September 2000 and as a senior strategic advisor to Ionis since January 2018. Previously she served as our chief operating officer from December 2007 through December 2017 and as chief financial officer from June 1994 through December2012.
IONIS EARNS $10 MILLION MILESTONE PAYMENT FROM BIOGEN FOR CARLSBAD, Calif., July 17, 2017 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) announced today that it has earned a $10 million milestone payment from Biogen associated with the validation of an undisclosed neurological disease target. Ionis and Biogen have a broad strategic collaboration to develop drugs to treat patients with neurological disorders. IONIS LICENSES NOVEL ANTISENSE DRUG FOR THE TREATMENT OF "CNM is a rare, debilitating disease affecting children and young adults and results in progressive muscular weakness. We are pleased that Dynacure has opted to license IONIS-DNM2-2.5 Rx, which we believe has the potential to bring benefit to patients with CNM," said Brett P. Monia, senior vice president of drug discovery and franchise leader for oncology and rare diseases at Ionis HOME | IONIS PHARMACEUTICALS, INC.ABOUTIONIS INNOVATIONOUR MEDICINESPATIENTS & COMMUNITYINVESTORS & MEDIACAREERS A perpetual and efficient innovation machine. Our drug discovery platform has served as a springboard for drug discovery and realized hope for patients with unmet needs. Our broad, diverse pipeline has more than 40 first-in-class and/or best-in-class medicines designed CAREERS | IONIS PHARMACEUTICALS, INC.INVESTOR FAQSCONTACTCOMMITTEE COMPOSITIONANTISENSE TECHNOLOGYOUR MEDICINES Ionis is where passionate, driven people come together to help patients in need. Our success is a direct result of our outstanding employees. Located in the North San Diego County city of Carlsbad, we’re an industry leader in the discovery and development of antisense drugs. Our work environment is challenging, motivating,rewarding, and
INVESTORS & MEDIA
Investors & Media. Ionis is focused on delivering RNA-targeted therapeutics with transformational potential. Our antisense oligonucleotide drug discovery platform has transformed the lives of patients with devastating diseases, including SMA, hATTR, and FCS. We have the potential to treat many additional diseases where othertherapeutic
ION373* | IONIS PHARMACEUTICALS, INC. ION373*. ION373 is an investigational antisense medicine targeting glial fibrillary acidic protein (GFAP) messenger ribonucleic acid (mRNA). ION373 is designed to inhibit the production of GFAP and is being developed as a potential therapy for Alexander disease (AxD). Nearly all cases of AxD are caused by gain-of-function mutations inGFAP that
OPTIMIZING ANTISENSE LICA, or Ligand Conjugated Antisense, is a chemical technology we developed at Ionis that involves the attachment of a molecule called a ligand that binds with receptors on the surfaces of cells in a highly specific manner. Because these receptors are often found only on certain cell types, LICA allows us to effectively deliver ourantisense
COMMUNITY ENGAGEMENT Corporate Alliances. Featured. 8/2. Surf Away SMA with Ricochet. La Jolla Shores Beach – Tower 33 La Jolla, CA 92037. In collaboration with Cure SMA, Ionis will continue to support local families effected by SMA for a wonderful event at the beach including food, fun, and surfing. With a limitless pursuit to support patients and familiesANNUAL REPORTS
The Investor Relations website contains information about Ionis Pharmaceuticals, Inc.'s business for stockholders, potential investors, and financial analysts. B. LYNNE PARSHALL, ESQ. B. Lynne Parshall, Esq. Senior Strategic Advisor, Ionis. Ms. Parshall has served on the Ionis board since September 2000 and as a senior strategic advisor to Ionis since January 2018. Previously she served as our chief operating officer from December 2007 through December 2017 and as chief financial officer from June 1994 through December2012.
IONIS EARNS $10 MILLION MILESTONE PAYMENT FROM BIOGEN FOR CARLSBAD, Calif., July 17, 2017 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) announced today that it has earned a $10 million milestone payment from Biogen associated with the validation of an undisclosed neurological disease target. Ionis and Biogen have a broad strategic collaboration to develop drugs to treat patients with neurological disorders. IONIS LICENSES NOVEL ANTISENSE DRUG FOR THE TREATMENT OF "CNM is a rare, debilitating disease affecting children and young adults and results in progressive muscular weakness. We are pleased that Dynacure has opted to license IONIS-DNM2-2.5 Rx, which we believe has the potential to bring benefit to patients with CNM," said Brett P. Monia, senior vice president of drug discovery and franchise leader for oncology and rare diseases at IonisOUR MEDICINES
A perpetual and efficient innovation machine. Our platform technology has served as a springboard for drug discovery and realized hope for patients with unmet needs. Our broad, diverse pipeline has more than 40 first-in-class and/or best-in-class medicines designed ABOUT | IONIS PHARMACEUTICALS, INC. Dr. Monia is the chief executive officer, a member of the board of directors and a founding scientist of Ionis Pharmaceuticals. His contributions at Ionis include research into the medicinal chemistry and mechanisms of action of RNA-targeting modalities to treat human diseases, most notably antisense-based therapeutic strategies. CONTACT US | IONIS PHARMACEUTICALS, INC. To report an Adverse Event or safety concern with use of an Ionis product, please call (800) 491-2664.If you are experiencing a medical emergency, please dial emergency services such as 911 or 112.PRESS RELEASES
Ionis' partner provides update on clinical studies evaluating tominersen (IONIS-HTT Rx) Mar 01, 2021. Ionis establishes new grant program to advance research and understanding of transthyretin amyloidosis. Feb 24, 2021. Ionis reports fourth quarter and full year 2020 financial results and recent business achievements. Feb 19, 2021. ION449 | IONIS PHARMACEUTICALS, INC. ION449. Chemistry: Generation 2.5 LICA. ION449 (formerly IONIS-AZ4-2.5-L Rx), also known as AZD8233, is a ligand-conjugated (LICA) investigational antisense medicine designed to reduce plasma levels of proprotein convertase subtilisin/kexin type 9 (PCSK9).PCSK9 is integrally involved in the regulation of LDL-cholesterol. Genetic studies have shown that individuals with life-long reduction of ANTISENSE TECHNOLOGY Antisense therapies are designed to seek out, bind to and destroy a mRNA in a highly specific manner, so that the amount of disease-causing protein is dramatically decreased. Antisense therapies can also treat diseases caused by too little protein by increasing the production of the protein, thereby restoring the protein to normallevels.
OPTIMIZING ANTISENSE LICA, or Ligand Conjugated Antisense, is a chemical technology we developed at Ionis that involves the attachment of a molecule called a ligand that binds with receptors on the surfaces of cells in a highly specific manner. Because these receptors are often found only on certain cell types, LICA allows us to effectively deliver ourantisense
TOFERSEN | IONIS PHARMACEUTICALS, INC. Tofersen. Chemistry: Generation 2 +. Tofersen (formerly IONIS-SOD1 Rx), also known as BIIB067, is an investigational antisense medicine targeting superoxide dismutase 1 (SOD1), which is the second most common and best understood genetic cause of amyotrophic lateral sclerosis (ALS).Patients with a mutation in the SOD1 gene develop a form of ALS referred to as SOD1-ALS. ION541* | IONIS PHARMACEUTICALS, INC. ION541, also known as BIIB105, is an investigational antisense medicine targeting ataxin 2 (ATXN2) RNA. ION541 is designed to reduce the production of ATXN2 protein. It is in development as a potential therapy for patients with most forms of Amyotrophic Lateral Sclerosis (ALS), regardless of family history. Aggregates of TDP-43 protein inmotor
BIOGEN AND IONIS EXPAND STRATEGIC COLLABORATION TO DEVELOP Transaction builds upon the productive collaboration that produced SPINRAZA ® as well as two antisense drug candidates currently in the clinic, with the potential to advance up to seven more drug candidates to the clinic within the next two years; The antisense platform developed by Ionis is an established and validated gene-based therapeutic modality, as evidenced by the success of SPINRAZASkip to content
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You are now leaving https://www.ionispharma.com to visit Continue to site CancelIONIS SAID YES
to discovering potential treatments for families living with Huntington’s Disease TRANSFORMATIVE TECHNOLOGY We are the leader in discovering and developing RNA-targeted therapeutics. Our efficient, broadly applicable drug discovery platform, called antisense technology, can treat illnesses where no other therapeutic approaches have proven effective or ever existed. Learn more about our Ionis Innovations ONCE IN A LIFETIME BREAKTHROUGHS AGAIN AND AGAIN We look to patients and their loved ones to help us better understand what it means to live with disease, so we can focus our efforts where we believe we can make the most meaningful contribution to improvelives.
See who inspires us
A PERPETUAL AND EFFICIENT INNOVATION MACHINE Our drug discovery platform has served as a springboard for drug discovery and realized hope for patients with unmet needs. Our broad, diverse pipeline has more than 40 first-in-class and/or best-in-class medicines designed to treat a broad range of diseases.Review our pipeline
PRESS RELEASES
View all press releasesMay
07
Akcea Expands Leadership Team with Appointment of Carla Poulson as Senior Vice President and Chief Human Resources Officerorange-arrowMay
06
Lancet Neurology: Teens and adults on SPINRAZA® showed clinically meaningful improvement in motor functionorange-arrowMay
06
Ionis reports first quarter 2020 financial results and recent business achievementsorange-arrowCAREERS
From the moment of our founding, we knew we could transform the biopharmaceutical industry. We had the people, the science and the unwavering commitment to bring about a new approach to drug discovery and development that would deliver innovative treatments to patients where no others had proven effective or ever existed. If you have this same commitment to be a force for life and feel that Ionis is right for you, we want to hear from you. Please be aware of employment scams using Ionis’ name and the names of Ionis employees to lure unsuspecting people into providing highly confidential and personal information. For more information click hereto read more.
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Please be aware of employment scams using Ionis’ name and the names of Ionis employees to lure unsuspecting people into providing highly confidential and personal information. For more information click hereto read more.
latest updates
Ionis recognized as one of the “World’s Top 10 Biotechnology Companies” by Investopedia Ionis establishes $25,000 scholarship to help ALS patients in greater San Diego area participate in physical and mental wellness programs Missed the live webinar "From The Gene to Treatment?" Watch thereplay here
Ionis and partner announce enrollment completion of global Phase 3 GENERATION HD1 study for Huntington's disease Ionis Pharmaceuticals first quarter 2020 financial results webcast - Watch the replay here Ionis Pharmaceuticals to host 2020 virtual annual meeting ofstockholders
Ionis reports first quarter 2020 financial results and recent business achievements Lancet Neurology: Teens and adults on SPINRAZA® showed clinically meaningful improvement in motor function Akcea Expands Leadership Team with Appointment of Carla Poulson as Senior Vice President and Chief Human Resources Officer Ionis Statement on COVID-19 1989 – 2020 Ionis PharmaceuticalsTM is a trademark of Ionis Pharmaceuticals, Inc. Akcea TherapeuticsTM is a trademark of Ionis Pharmaceuticals, Inc.* Contact
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