gene therapy – Talk Bio

uniQure Faces Delay as FDA Walks Back Accelerated Approval for Huntington’s Gene Therapy

6 days ago talkbio0Tagged Accelerated, AMT-130, Approved, Clinical Trials, gene therapy, Huntington 's disease, Regulatory Setback, UniQure, United States Food and Drug Administration

uniQure; AMT-130; Huntington’s disease; gene therapy; FDA; accelerated approval; regulatory setback; clinical trial

Otsuka Acquires Asia-Pacific Rights to 4D Molecular Therapeutics’ Retinal Gene Therapy 4D-150 for $85 Million

1 week ago talkbio0Tagged 4D Molecular Therapeutics ', Asia-Pacific, gene therapy, Macular edema due to diabetes mellitus, Ophthalmology specialty, Otsuka, strategic partnership, Wet Age-Related Macular Degeneration

Otsuka; 4D Molecular Therapeutics; 4D-150; Asia-Pacific rights; gene therapy; wet age-related macular degeneration; diabetic macular edema; $85 million; strategic partnership; ophthalmology

FDA Places Clinical Hold on Two Intellia CRISPR Trials After Life-Threatening Liver Toxicity Case

1 week ago talkbio0Tagged ATTR-CM, ATTR-PN, Clustered Regularly Interspaced Short Palindromic Repeats, gene therapy, Intellia, Liver, MAGNITUDE trial, Nexiguran, Senile cardiac amyloidosis, Study on Hold, Toxicity aspects, United States Food and Drug Administration, ziclumeran

FDA; Intellia; CRISPR; clinical hold; liver toxicity; nexiguran ziclumeran; MAGNITUDE trial; transthyretin amyloidosis; ATTR-CM; ATTR-PN; gene therapy

BioMarin Plans to Divest Hemophilia Gene Therapy Roctavian Amid Strategic Refocus

2 weeks ago talkbio0Tagged BioMarin, Divestment, gene therapy, Hemophilia A, out-licensing, portfolio strategy, Roctavian

BioMarin; Roctavian; hemophilia A; gene therapy; divestment; out-licensing; portfolio strategy; third quarter 2025; revenue challenge

Lilly Expands Gene Therapy Presence with Acquisition of Adverum Biotechnologies

2 weeks ago talkbio0Tagged Acquisition, Adverum Biotechnologies, contingent value, eli lilly, gene therapy, ixovec, Ophthalmology specialty, Wet Age-Related Macular Degeneration

Eli Lilly; Adverum Biotechnologies; gene therapy; ophthalmology; wet age-related macular degeneration; ixovec; acquisition; contingent value rights

Roche in Talks With US on Drug Pricing; Sets Out Pipeline and Dealmaking Ambitions

3 weeks ago talkbio0Tagged Benefit, dealmaking, direct-to-consumer sales, drug price, gene therapy, Government, Hemlibra, Investments, manager, Manufacturing, Ocrevus, Pharmaceutical Services, pipeline, R&D, Roche (company), U.S., Vabysmo

Roche; drug pricing; US government; pharmacy benefit managers; direct-to-consumer sales; pipeline; dealmaking; US investment; manufacturing; gene therapy; R&D; Ocrevus; Hemlibra; Vabysmo

Diorasis Therapeutics Welcomes Dr. Dimitri Azar to Its Board of Directors

3 weeks ago talkbio0Tagged Biotechnology, board of directors, Clinical Development, Dimitri Azar, Diorasis, gene therapy, Glaucoma, Ophthalmology specialty, United States Food and Drug Administration

Diorasis Therapeutics; Dimitri Azar; Board of Directors; gene therapy; glaucoma; ophthalmology; biotechnology; FDA; clinical development

Recent Advances and News in In Vivo Therapeutic Technologies (2025)

3 weeks ago talkbio0Tagged Acquisitions, Autoimmune Diseases, CAR-T, Cell Therapy, drug delivery innovation, Fast Track, gene therapy, in vivo, nanoparticle delivery, Neoplasms, RNA delivery, United States Food and Drug Administration

in vivo CAR-T; cell therapy; gene therapy; RNA delivery; autoimmune disease; oncology; nanoparticle delivery; drug delivery innovation; FDA fast track; biotech acquisitions

Krystal Biotech Receives FDA Platform Technology Designation for HSV-1 Viral Vector Used in KB801 for Neurotrophic Keratitis

3 weeks ago talkbio0Tagged gene therapy, HSV-1 viral vector, KB801, Krystal Biotech, Neurotrophic keratitis, platform technology designation, redesable eye drop, United States Food and Drug Administration, VYJUVEK

Krystal Biotech; FDA; platform technology designation; HSV-1 viral vector; KB801; gene therapy; neurotrophic keratitis; redesable eye drop; VYJUVEK

Gene Therapy Delivers Long-Term Immune Protection in Children With Rare Immune Disorders

4 weeks ago talkbio0Tagged Adenosine deaminase deficiency, Clinical Trials, Combined immunodeficiency disease, Congenital leukocyte adherence deficiency, Curative Therapy, gene therapy, immune protection, Leukocyte adhesion deficiency type 1, Long-term, Pediatric immunology, Rare Diseases, Severe (severity modifier)

gene therapy; long-term immune protection; ADA-SCID; severe combined immunodeficiency; LAD-I; leukocyte adhesion deficiency; rare disease; pediatric immunology; clinical trial; curative treatment