gene therapy – Talk Bio

Biotech Leaders Urge FDA to ‘Modernise’ Regulation to Compete with China

2 days ago talkbio0Tagged Cells, CEO, China, competitiveness, gene therapy, innovation, Modernization, Regulation, roundtable, United States Food and Drug Administration

FDA; biotech; regulation; modernization; China; cell and gene therapy; CEO roundtable; competitiveness; innovation

Regenxbio Reports Consistent One-Year Benefit for DMD Gene Therapy RGX-202

2 days ago talkbio0Tagged AFFINITY DUCHENNE trial, Biological Markers, Clinical Trials, Functional, gene therapy, Muscular Dystrophy, Duchenne, Phase III, Regenxbio, SLC6A8 Inhibitor RGX-202

Regenxbio; DMD; Duchenne muscular dystrophy; RGX-202; gene therapy; AFFINITY DUCHENNE trial; clinical trial; microdystrophin; biomarker; phase I/II; functional data

Sarepta’s rAAVrh74 Viral Vector Receives FDA Platform Technology Designation for Gene Therapy

4 days ago talkbio0Tagged gene therapy, Infrequent, Muscular Dystrophies, Limb-Girdle, platform technology designation, SRP-9003, United States Food and Drug Administration, Viral Vector

Sarepta Therapeutics; FDA; platform technology designation; rAAVrh74 viral vector; gene therapy; SRP-9003; limb-girdle muscular dystrophy; rare diseases

Biopharma Briefing: Q1 Trends and Updates

5 days ago talkbio0Tagged ASCO preview, Biopharma, gene therapy, Q1 trends

biopharma; Q1 trends; gene therapy; ASCO preview

Analysts Downplay Intellia Adverse Event, but Gene Therapy Safety Remains Under Scrutiny

1 week ago talkbio0Tagged Adverse event, ATTR-CM, Event, Gene Modification, gene therapy, Intellia, liver transaminase, MAGNITUDE trial, Response process, Safety, Stock Decline

Intellia Therapeutics; gene therapy; safety event; adverse event; MAGNITUDE trial; liver transaminase; stock decline; ATTR-CM; gene editing; analyst response

Patient Death in Rocket Pharmaceuticals Gene Therapy Trial Prompts FDA Clinical Hold

2 weeks ago talkbio0Tagged acute systemic infection, Adverse event, Capillary Leak Syndrome, gene therapy, Glycogen Storage Disease Type IIb, Patient Death, Rocket, RP-A501, Study on Hold, United States Food and Drug Administration

Rocket Pharmaceuticals; gene therapy; Danon disease; clinical hold; FDA; patient death; capillary leak syndrome; acute systemic infection; RP-A501; adverse event

RegenXBio Secures Up to $250 Million by Monetizing Royalties from Zolgensma and Two Gene Therapies

3 weeks ago talkbio0Tagged gene therapy, HealthCare Royalty, milestone payments, non-dilutive financing, Regenxbio, RGX-111, RGX-121, royalty monetization, Zolgensma

RegenXBio; Zolgensma; gene therapy; royalty monetization; HealthCare Royalty; RGX-121; RGX-111; non-dilutive financing; biotech; milestone payments

Prime Medicine CEO Exits Amidst Layoffs and Restructuring as Sole Clinical Gene Therapy Shelved

3 weeks ago talkbio0Tagged CEO, Clinical Trials, Exit, gene therapy, layoffs, pipeline pivot, PM359, Prime Editing, Prime Medicine, restructuring

Prime Medicine; CEO exit; gene therapy; layoffs; restructuring; biotech; PM359; prime editing; clinical trial; pipeline pivot

Eli Lilly Expands RNA Pipeline with $1.3 Billion Rznomics Hearing Loss Pact

3 weeks ago talkbio0Tagged Deafness, Eli, Eli Lilly, gene therapy, pharmaceutical partnerships, RNA Editing, RNA therapeutics, Rznomics, Sensorineural Hearing Loss (disorder), trans-splicing ribozyme

Eli Lilly; Rznomics; RNA editing; hearing loss; gene therapy; pharmaceutical partnerships; biotech; trans-splicing ribozyme; sensorineural hearing loss; RNA therapeutics

Neurogene Details New Safety Measures After Young Patient’s Death in Rett Syndrome AAV Gene Therapy Trial

3 weeks ago talkbio0Tagged AAVS1 gene, ASGCT, Clinical Trials, Dosage, gene therapy, Individual Adjustment, Lymphohistiocytosis, Hemophagocytic, Measures, MECP2 gene, Neurogene, NGN-401, Rett Syndrome, Safety, United States Food and Drug Administration

Neurogene; AAV gene therapy; Rett syndrome; NGN-401; ASGCT; safety measures; HLH; clinical trial; MECP2 gene; FDA; dose adjustment