SAN FRANCISCO, Calif. — Invitae (NYSE: NVTA), a leading medical genetics company, today announced a partnership with BridgeBio Pharma, Inc. (Nasdaq: BBIO), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, designed to advance genetics-based drug discovery for rare diseases. The goal of the collaboration is to generate new insights...
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NEW YORK, NY — IO Biotech (Nasdaq: IOBT), a clinical-stage biopharmaceutical company developing novel, immune-modulatory, off-the-shelf therapeutic cancer vaccines, today announced the publication in Nature Communications of the long-term clinical and immunological outcomes from MM1636, the Phase 1/2 trial evaluating the investigational peptide vaccine IO102-IO103 targeting both tumor cells and immune-suppressive...
GENEVA, Switzerland and AMSTERDAM, The Netherlands — iOnctura, a clinical-stage biotech developing selective cancer therapies against targets that play critical roles in multiple tumor survival pathways, today announces that the US Food and Drug Administration (FDA) has granted permission to proceed with clinical investigations of roginolisib in the United States...
CARLSBAD, Calif. and CAMBRIDGE, Mass. — Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) and Biogen Inc. (Nasdaq: BIIB) announced the decision to terminate development of BIIB105 (ION541) an investigational antisense oligonucleotide (ASO) for amyotrophic lateral sclerosis (ALS) based on topline results from the Phase 1/2 ALSpire study. BIIB105 was designed to reduce expression of...
CARLSBAD, Calif. — Ionis Pharmaceuticals, Inc. today announced that it has entered into a license agreement with Otsuka Pharmaceutical Co., Ltd. under which Otsuka obtains exclusive rights in Europe to commercialize donidalorsen, an investigational prophylactic treatment for hereditary angioedema (HAE). Ionis will maintain responsibility for the non-clinical and clinical development...
CARLSBAD, Calif. — Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced positive topline data from the HALOS Phase 1/2a open-label study of ION582 in Angelman syndrome. ION582 was safe and well tolerated in the study and showed encouraging and consistent benefits in individuals living with Angelman syndrome, with the most robust improvements observed...
CARLSBAD, Calif. — Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced positive topline results from the pivotal study of zilganersen in children and adults living with Alexander disease (AxD), a rare, progressive and often fatal neurological condition with no approved disease-modifying treatments. Zilganersen 50 mg demonstrated statistically significant and clinically meaningful stabilization...
CARLSBAD, Calif. – Ionis Pharmaceuticals (NASDAQ: IONS) today announced that the company has launched a new grant program that provides funding to U.S.-based researchers whose work advances the understanding of transthyretin amyloidosis (ATTR), facilitates early diagnosis and enhances disease management of patients living with ATTR amyloidosis. The Janice Wiesman Young...
CARLSBAD, Calif. – Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), the leader in RNA-targeted therapies, announced today initiation of OASIS-HAE, the registrational study in the donidalorsen (formerly IONIS-PKK-LRx) Phase 3 clinical program. Donidalorsen is an investigational antisense medicine designed to reduce the production of prekallikrein, which plays a key role in the activation...
CARLSBAD, Calif. – Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) announced the initiation of a Phase 3 clinical trial of ION363 in patients with amyotrophic lateral sclerosis (ALS) with mutations in the fused in sarcoma gene (FUS). Patients with a mutation in the FUS gene develop a rare form of ALS, referred...