Disease or Syndrome – Page 32

Abivax aces pair of phase 3 ulcerative colitis trials as end of cash runway looms

4 months ago talkbio0Tagged Abivax, ABTECT-1, ABTECT-2, cash runway, clinical remission, induction trials, maintenance trial, Obefazimod, Oral cavity, Ulcerative Colitis

Abivax; Obefazimod; ulcerative colitis; Phase 3 trial; clinical remission; ABTECT-1; ABTECT-2; cash runway; induction trials; oral miR-124 enhancer; maintenance trial

Sarepta Therapeutics Faces FDA Crackdown after Third Patient Death; George Tidmarsh Named Top FDA Drug Regulator; Multiple Drugs Rejected; Pharma Invests Billions in US Manufacturing

4 months ago talkbio0Tagged drug rejection, ELEVIDYS, gene therapy, George Tidmarsh, Liver Failure, Acute, pharmaceutical manufacturing, reshoring, Study on Hold, United States Food and Drug Administration

Sarepta Therapeutics; FDA; gene therapy; Elevidys; acute liver failure; clinical hold; George Tidmarsh; drug rejection; reshoring; pharmaceutical manufacturing

Lack of Transparency Tarnishes Sarepta’s Sheen as Patient Deaths Trigger FDA Battle

4 months ago talkbio0Tagged ELEVIDYS, gene therapy, lack of transparency, Market, Muscular Dystrophies, Limb-Girdle, Muscular Dystrophy, Duchenne, patient deaths, Regulatory Action, United States Food and Drug Administration, Withdraw (activity)

Sarepta Therapeutics; Elevidys; patient deaths; FDA; lack of transparency; gene therapy; market withdrawal; regulatory action; limb-girdle muscular dystrophy (LGMD); Duchenne muscular dystrophy

Sarepta, bowing to FDA pressure, pauses shipments of Duchenne gene therapy Elevidys

4 months ago talkbio0Tagged ELEVIDYS, gene therapy, Muscular Dystrophy, Duchenne, patient deaths, Pauses, Safety, shipment, United States Food and Drug Administration

Sarepta Therapeutics; FDA; Elevidys; Duchenne muscular dystrophy; gene therapy; shipment pause; patient deaths; safety concerns

Avalyn Pharma Raises $100M to Advance Inhaled Lung Disease Drugs

4 months ago talkbio0Tagged AP01, AP02, aspects of adverse effects, Avalyn, Clinical Trials, drug safety, Holdings, inhaled lung disease, nintedanib, Pharma, pirfenidone, Pulmonary Fibrosis, Series D, SNCG gene, Suvretta Capital Management

Avalyn Pharma; $100 million Series D; inhaled lung disease medications; pulmonary fibrosis; AP01; AP02; pirfenidone; nintedanib; clinical trials; drug safety; side effects; Suvretta Capital Management; SR One; Novo Holdings

Replimune’s Shares Plunge After FDA Rejects Melanoma Drug Application

4 months ago talkbio0Tagged Antineoplastic Agents, Clinical Trial Design, Complete, Drops - Drug Form, Frequency of Responses, melanoma, Prices, Rejection, Replimune, Response Letter, RETINITIS PIGMENTOSA 1, United States Food and Drug Administration

Replimune; RP1; FDA rejection; melanoma; cancer drug; Complete Response Letter; clinical trial design; objective response rate; share price drop

AstraZeneca Unveils $50 Billion U.S. Investment, Including Major Manufacturing Facility in Virginia

4 months ago talkbio0Tagged AstraZeneca, Chronic disease, Investments, Manufacturing, R&D, Surgical construction, tariffs, United States, Virginia plant

AstraZeneca; investment; United States; Virginia plant; manufacturing; R&D; tariffs; chronic diseases; job creation; pharmaceuticals

Alkermes Advances Narcolepsy Therapy with Promising Phase 2 Trial Results for Alixorexton

4 months ago talkbio0Tagged alixorexton, Alkermes, Cataplexy, Daytime Somnolence, Excessive (qualifier value), late-stage testing, Narcolepsy, Narcolepsy type 1, normative wakefulness, Orexin Receptor Agonist, Vibrance-1 study

Alkermes; narcolepsy; alixorexton; Phase 2 trial; orexin 2 receptor agonist; narcolepsy type 1; normative wakefulness; late-stage testing; Vibrance-1 study; cataplexy; excessive daytime sleepiness

Sarepta Stands Behind Elevidys After FDA Requests Withdrawal Amid Safety Concerns

4 months ago talkbio0Tagged Cessation of life, Clinical Trials, concerns, ELEVIDYS, gene therapy, Market, Muscular Dystrophy, Duchenne, Safety, United States Food and Drug Administration, Withdraw (activity)

Sarepta; Elevidys; FDA; gene therapy; market withdrawal; safety concerns; Duchenne muscular dystrophy; deaths; clinical trial hold

Biogen Pledges $2 Billion Manufacturing Investment in North Carolina

4 months ago talkbio0Tagged Antisense Oligonucleotides, Artificial Intelligence, Automation, Biogen, Biotechnology, expansion, gene therapy, Investments, Manufacturing, North Carolina, Research Triangle Park

Biogen; manufacturing investment; North Carolina; Research Triangle Park; biotechnology; antisense oligonucleotides; gene therapy; automation; AI; expansion