gene therapy – Page 3

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

2 months 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

Regeneron Heads to FDA Seeking Approval for Game-Changing Hearing Loss Gene Therapy

2 months ago talkbio0Tagged Approved, CHORD trial, DB-OTO, Deafness, gene therapy, OTOF gene, Regeneron, United States Food and Drug Administration

Regeneron; hearing loss; gene therapy; DB-OTO; FDA approval; OTOF gene; CHORD trial

Patient with Preexisting Immunity to AAV Vectors Granted Gene Therapy After Antibody-Depleting Agent Allowed Eligibility

2 months ago talkbio0Tagged AAV antibodies, antibody-chopping drug, Clinical Trials, Eligibility Determination, gene therapy

gene therapy; AAV antibodies; ineligible; antibody-chopping drug; clinical trial; eligibility

AviadoBio Secures Option to Develop Rare Eye Disease Gene Therapy UGX-202 in $413 Million Deal with China’s UgeneX

2 months ago talkbio0Tagged AviadoBio, China, gene therapy, Optogenetics, Retinitis Pigmentosa, UgeneX, UGX-202

AviadoBio; UgeneX; UGX-202; optogenetics; gene therapy; retinitis pigmentosa; China; $413 million deal

AviadoBio Secures Option to UgeneX’s UGX-202 Gene Therapy for Retinal Disease

2 months ago talkbio0Tagged AviadoBio, Clinical Development, Dependovirus, gene therapy, Optogenetics, Retinal Diseases, Retinitis Pigmentosa, UGX-202

AviadoBio; UgeneX Therapeutics; UGX-202; retinitis pigmentosa; gene therapy; optogenetics; adeno-associated virus (AAV); exclusive option; retinal disease; clinical development

Rocket Pharmaceuticals Withdraws FDA Application for Rare Blood Disease Gene Therapy

2 months ago talkbio0Tagged Fanconi Anemia, gene therapy, Mozafancogene Autotemcel, pipeline pivot, Rocket Pharmaceuticals, United States Food and Drug Administration

Rocket Pharmaceuticals; FDA; RP-L102; Fanconi Anemia; Gene Therapy; Pipeline Pivot

Meeting on the Mesa 2025 Arrives Amid Mixed Signals for Cell and Gene Therapy Sector

2 months ago talkbio0Tagged Advanced therapies, Alternative, Cells, Clinical Trials, commercialization, Gene Modification, gene therapy, Industry, meeting, MESA, partnerships, payment, Phoenix Arizona, regulatory challenges, RNA, Messenger, Study models

Cell and gene therapy; Meeting on the Mesa; Commercialization; Clinical trials; Advanced therapies; Industry partnerships; Regulatory challenges; Phoenix Arizona; mRNA; Gene editing; Alternative payment models