gene therapy – Page 5

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

3 months 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 months 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 months 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 months 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

Blood cancer biotech Vor Bio ends clinical work, lays off 95% of staff

4 months ago talkbio0Tagged 95%, gene therapy, Hematologic Neoplasms, Leukemia, Myelocytic, Acute, Reflex, Vestibulo-Ocular

Hematologic Neoplasms, Leukemia, Myelocytic, Acute, Reflex, Vestibulo-Ocular, gene therapy, 95%

Beacon’s rare eye disease gene therapy signals vision function improvements in early phase 2 data

4 months ago talkbio0Tagged DAWN, Disorder of eye, gene therapy, laru-zova, Phase 2 trial, Vision, XLRP

XLRP, gene therapy, laru-zova, visual function, rare eye disease, Phase 2 trial, DAWN trial, vision improvement, low light vision

J&J’s $130M gene therapy bet flunks rare retinal disease trial

4 months ago talkbio0Tagged bota-vec, Disorder of eye, Failure (biologic function), gene therapy, Infrequent, Investments, Johnson and Johnson, loss treatment, Phase 3, Vision, X-linked retinitis pigmentosa

Johnson & Johnson, gene therapy, bota-vec, X-linked retinitis pigmentosa, Phase 3 failure, vision loss treatment, rare eye disease, $130 million investment

Exclusive: Vertex drops AAV gene therapy research, tRNA therapy partnership

4 months ago talkbio0Tagged Dependovirus, gene therapy, Industry, Partnership, research, Safety, Transfer RNA

gene therapy, Dependovirus, Transfer RNA, Partnership, Industry, research, Safety

Abeona’s $3.1M gene therapy wins FDA nod for rare skin disease

4 months ago talkbio0Tagged Abeona, Abeona 's, Approved, Dermatologic disorders, gene therapy, Hallopeau-Siemens Disease, Infrequent, United States Food and Drug Administration, ZEVASKYN

United States Food and Drug Administration, United States Food and Drug Administration, Infrequent, ZEVASKYN, Abeona, gene therapy, Dermatologic disorders, Hallopeau-Siemens Disease, Approved, Abeona ‘s

FDA approves Abeonas skin disease cell therapy

4 months ago talkbio0Tagged Abeona, Approved, gene therapy, Hallopeau-Siemens Disease, Treating, United States Food and Drug Administration, Wounds - qualifier, ZEVASKYN

ZEVASKYN, United States Food and Drug Administration, Hallopeau-Siemens Disease, Approved, Wounds – qualifier, gene therapy, Abeona, Treating