ENTITY – Page 7

FDA Rejection Derails PTC Therapeutics’ Rare Disease Hopeful

2 weeks ago talkbio0Tagged Clinical Trials, concerns, Effectiveness, factor IX, Failure (biologic function), Friedreich 's ataxia, Rare Diseases, Rejection, Response process, United States Food and Drug Administration, vatiquinone

PTC Therapeutics; FDA rejection; vatiquinone; Friedreich’s ataxia; rare disease; efficacy concerns; complete response letter; clinical trial failure

FDA Extends PDUFA Date for Regenxbio’s Hunter Syndrome Gene Therapy RGX-121

2 weeks ago talkbio0Tagged clemidsogene lanparvovec, Deferred, gene therapy, IDS gene, Infrequent, Mucopolysaccharidosis II, PDUFA, Regenxbio, RGX-121, United States Food and Drug Administration

FDA delay; PDUFA extension; Regenxbio; Hunter syndrome; clemidsogene lanparvovec; RGX-121; gene therapy; Mucopolysaccharidosis II; IDS gene; rare disease

VantAI and Halda Strike $1B+ AI-Driven Partnership on ‘Hold-and-Kill’ Drugs

2 weeks ago talkbio0Tagged Aortic Valve Insufficiency, cellular targeting, Chimera organism, Drug Discovery, Halda Therapeutics, hold-and-kill, Immunology, Induce (action), Neoplasms, Proximity, RIPTAC, target-effector pairs, VantAI

VantAI; Halda Therapeutics; RIPTAC drugs; hold-and-kill; AI drug discovery; target-effector pairs; oncology; immunology; regulated induced proximity targeting chimeras; $1 billion deal

Shankar Ramaswamy’s Kriya Therapeutics Raises $313M for Gene Therapy Advancements

2 weeks ago talkbio0Tagged Biotechnology, gene therapy, Kriya, Shankar Ramaswamy, venture

Kriya Therapeutics; Shankar Ramaswamy; gene therapy; biotech funding; fundraising; biotechnology; venture capital; 2025

Battle Intensifies in the Obesity Drug Market as Novo Nordisk and Eli Lilly Vie for Dominance

2 weeks ago talkbio0Tagged Benefit, Clinical Trials, competition, Compounders, Direct-to-Consumer, eli lilly, Glucagon-Like Peptide 1, manager, Market, Nordisk, Obesity, Pharmaceutical Services, Wegovy, Zepbound

obesity drugs; GLP-1; Wegovy; Zepbound; Novo Nordisk; Eli Lilly; market competition; direct-to-consumer; pharmacy benefit managers; compounders; clinical trials

Stealth BioTherapeutics Resubmits Third FDA Application for Rare Disease Drug

2 weeks ago talkbio0Tagged 3-Methylglutaconic aciduria type 2, access program, Drug Approval, Elamipretide, Infrequent, resubmission, Stealth BioTherapeutics, United States Food and Drug Administration

Stealth BioTherapeutics; FDA resubmission; rare disease; Barth syndrome; elamipretide; drug approval; expanded access program

Merck KGaA and Skyhawk Therapeutics Launch $2B RNA-Targeting Neurology Collaboration

2 weeks ago talkbio0Tagged Billion Collaboration, Drug Development, Merck KGaA, milestone payments, nervous system disorder, RNA-targeting, royalties, Skyhawk, SkySTAR platform

Merck KGaA; Skyhawk Therapeutics; RNA-targeting; SkySTAR platform; neurological disorders; $2 billion collaboration; drug development; milestone payments; royalties

Federal Appeals Court Overturns FDA Rejection of Vanda’s Hetlioz for Jet Lag Disorder

2 weeks ago talkbio0Tagged Clinical Trials, Evidence, Federal Appeals Court, Hetlioz, jet lag disorder, New Drug Application, regulatory law, sNDA, United States Food and Drug Administration, Vanda Pharmaceuticals

Vanda Pharmaceuticals; Hetlioz; FDA; jet lag disorder; Federal Appeals Court; supplemental New Drug Application (sNDA); regulatory law; clinical trial evidence

Reunion Psychedelic Therapy Advances to Pivotal Trials after Postpartum Depression Success

2 weeks ago talkbio0Tagged Clinical Trials, Depression, Postpartum, Montgomery-Åsberg Depression Rating Scale, Montgomery-Asberg Depression Rating Scale Questionnaire, Neuroscience discipline, Phase 2 Clinical Trials, Phase 3 Clinical Trials, psychedelic therapy, RE104, RECONNECT, remission rates, Reunion

Reunion Neuroscience; RE104; psychedelic therapy; postpartum depression (PPD); Phase II trial; Phase III trial; RECONNECT trial; clinical trial results; Montgomery-Åsberg Depression Rating Scale (MADRS); remission rates

Conflicts of Interest Among Federal Vaccine Advisors at Historic Lows Through 2024

2 weeks ago talkbio0Tagged ACIP, Advisory Committees, Centers for Disease Control and Prevention (U.S.), JAMA study, Robert F. Kennedy Jr., Translucent, United States Food and Drug Administration, Vaccines, VRBPAC

conflicts of interest; vaccine advisory committees; ACIP; VRBPAC; CDC; FDA; transparency; Robert F. Kennedy Jr.; JAMA study