Browse News

Found 569 articles

• Dyne Therapeutics Receives European Medicines Agency (EMA) Orphan Drug Designation for DYNE-101

  5/25/2023

  Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases, today announced that the European Medicines Agency (EMA) has granted orphan drug designation for DYNE-101.

• 

  Wave’s Antisense ALS Therapy Is Company’s Latest Miss

  5/24/2023

  Wave will discontinue development of WVE-004 in C9-associated ALS and frontotemporal dementia after the therapy failed to show any clinical benefit in a mid-stage trial.

• Wave Life Sciences Announces Topline Results from Phase 1b/2a FOCUS-C9 Study of WVE-004 for C9orf72-associated Amyotrophic Lateral Sclerosis and Frontotemporal Dementia

  5/23/2023

  Wave Life Sciences Ltd. today announced topline results from the Phase 1b/2a FOCUS-C9 study evaluating WVE-004 as an investigational treatment for C9orf72-associated amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) (C9-ALS/FTD).

• Dyne Therapeutics’ Preclinical Data Demonstrating FORCE™ Platform Delivery to CNS Featured in Oral Presentation at ASGCT Annual Meeting

  5/17/2023

  Dyne Therapeutics, Inc. is delivering an oral presentation today at the American Society of Gene & Cell Therapy (ASGCT) 26th Annual Meeting in Los Angeles highlighting new preclinical data demonstrating the FORCE™ platform achieved delivery to the central nervous system (CNS) in non-human primates and robust pharmacological effects in the brain in a model of myotonic dystrophy type 1 (DM1).

• Chinook Therapeutics Announces Partnership with Ionis to Develop Antisense Therapy for Rare, Severe Chronic Kidney Disease

  5/16/2023

  Chinook Therapeutics, Inc. (Nasdaq: KDNY) today announced it has entered a collaboration agreement with Ionis Pharmaceuticals, Inc. for the discovery, development and commercialization of an antisense oligonucleotide (ASO) therapy for a rare, severe chronic kidney disease with significant unmet medical need.

• Praxis Precision Medicines Announces Positive Topline Results from PRAX-628 Phase 1 Study Enabling Best-in-Class Profile

  5/11/2023

  Praxis Precision Medicines, Inc. announced positive topline results from the PRAX-628 Phase 1 healthy volunteer study evaluating the safety, tolerability and pharmacokinetics of single ascending doses and multiple ascending doses of PRAX-628.

• Praxis Precision Medicines Provides Corporate Update and Reports First Quarter 2023 Financial Results

  5/11/2023

  Praxis Precision Medicines, Inc. (NASDAQ: PRAX), a clinical-stage biopharmaceutical company translating genetic insights into the development of therapies for central nervous system (CNS) disorders characterized by neuronal excitation-inhibition imbalance, today provided a corporate update and reported financial results for the first quarter 2023.

• Denali Therapeutics Reports First Quarter 2023 Financial Results and Business Highlights

  5/8/2023

  Denali Therapeutics Inc. (Nasdaq: DNLI) today reported financial results for the first quarter ended March 31, 2023, and provided business highlights.

• Suzhou GenAssist Therapeutic Co.,Ltd recently announced that its first base editing product, GEN6050, has submitted a pre-IND application to the FDA and has been accepted

  5/6/2023

  Suzhou GenAssist Therapeutic Co.,Ltd recently announced its pre-IND application of their first base editing product, GEN6050 and the acceptance by the FDA.

• Stoke Therapeutics Reports First Quarter Financial Results and Provides Business Updates - May 4, 2023

  5/4/2023

  Stoke Therapeutics, Inc. today reported financial results for the first quarter of 2023 and provided business updates including those related to STK-001, the company’s proprietary antisense oligonucleotide (ASO) being developed by Stoke as the first potential new medicine to address the genetic cause of Dravet syndrome.

• Aligos Therapeutics Reports Recent Business Progress and First Quarter 2023 Financial Results

  5/4/2023

  Aligos Therapeutics, Inc. reported recent business progress and financial results for the first quarter 2023.

• Stoke Therapeutics to Present at the BofA Securities Health Care Conference

  5/3/2023

  Stoke Therapeutics, Inc. today announced that management will present at the BofA Securities Health Care Conference on Thursday, May 11, 2023, at 11:00 a.m. ET. (8:00 a.m. PT).

• Evolent Health Announces First Quarter 2023 Results

  5/3/2023

  Evolent Health, Inc. (NYSE: EVH), a health care company that delivers proven clinical and administrative solutions to payers and providers, today announced financial results for the quarter ended March 31, 2023.

• Dyne Therapeutics to Present New Preclinical Data at ASGCT Annual Meeting Demonstrating FORCE™ Platform Delivery to CNS

  5/2/2023

  Dyne Therapeutics, Inc. (Nasdaq: DYN) today announced that new preclinical data demonstrating the FORCE™ platform achieved delivery to the central nervous system (CNS) and robust pharmacological effects in the brain will be featured in an oral presentation at the American Society of Gene & Cell Therapy (ASGCT) 26th Annual Meeting.

• FDA approves QALSODY™ (tofersen) as the first treatment targeting a genetic cause of ALS

  4/25/2023

  Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that its partner Biogen has received U.S. Food and Drug Administration (FDA) approval of QALSODY™ (tofersen) 100 mg/15mL injection for the treatment of amyotrophic lateral sclerosis (ALS) in adults who have a mutation in the superoxide dismutase 1 (SOD1) gene.

• BIORCHESTRA Scientific Founder, Chairman, and Chief Executive Officer Joins the Republic of Korea (ROK) Official State Visit to the United States

  4/25/2023

  BIORCHESTRA announced that the company's Scientific Founder, Chairman, and Chief Executive Officer, Dr. Branden Ryu, has been invited by the Korean Government to join the Official State visit to the U.S.

• Muscular Dystrophy Association Celebrates FDA Approval of Biogen’s Qalsody for Treatment of SOD1-ALS

  4/25/2023

  The Muscular Dystrophy Association (MDA) celebrates the US Food and Drug Administration (FDA) accelerated approval of Qalsody (tofersen), for the treatment of amyotrophic lateral sclerosis (ALS, also known as Lou Gehrig’s disease) associated with mutation in the superoxide dismutase 1 (SOD1) gene (SOD1-ALS).

• FDA Grants Accelerated Approval for QALSODY™ (tofersen) for SOD1-ALS, a Major Scientific Advancement as the First Treatment to Target a Genetic Cause of ALS

  4/25/2023

  Biogen Inc. (Nasdaq: BIIB) announced today that the U.S. Food and Drug Administration (FDA) has approved QALSODY™ (tofersen) 100 mg/15mL injection for the treatment of amyotrophic lateral sclerosis (ALS) in adults who have a mutation in the superoxide dismutase 1 (SOD1) gene.

• Stoke Therapeutics Receives Authorization to Initiate a Phase 1/2 Study of STK-002 for Autosomal Dominant Optic Atrophy (ADOA) in the United Kingdom

  4/25/2023

  Stoke Therapeutics, Inc. today announced authorization of its Clinical Trial Application (CTA) by the United Kingdom Medicines and Healthcare products Regulatory Agency (MHRA) to initiate a Phase 1/2 study (OSPREY) of STK-002 for the treatment of autosomal dominant optic atrophy (ADOA), the most common inherited optic nerve disorder.

• BIORCHESTRA Announces Significant Progress in Studies of BMD-001 Drug Program

  4/24/2023

  BIORCHESTRA, a biotherapeutics company focused on rare and degenerative diseases within the Central Nervous System (CNS), today announced significant progress in its lead drug program – BMD-001.