gene therapy – Page 4

Recent News: Gain Peace of Mind with the Lentiviral Experts

3 months ago talkbio0Tagged advanced manufacturing, Biological Factors, CAR-T therapy, gene therapy, Growth, immuno-oncology, lentiviral vectors, Market, regulatory approval

lentiviral vectors; CAR-T therapy; gene therapy; biologics; immuno-oncology; market growth; advanced manufacturing; regulatory approval

bluebird bio Rebrands as Genetix Biotherapeutics Following Private Equity Buyout

3 months ago talkbio0Tagged Anemia, Sickle Cell, Bluebird Bio, Capital Partners, Carlyle, Cerebral Adrenoleukodystrophy, commercial, David Meek, DKK1 gene, execution, FDA-approved therapies, gene therapy, Genetix Biotherapeutics, Investments, Manufacturing, Private Equity, rebranding, Thalassemia

bluebird bio; Genetix Biotherapeutics; private equity buyout; Carlyle; SK Capital Partners; gene therapy; rebranding; FDA-approved therapies; David Meek; sickle cell disease; β-thalassemia; cerebral adrenoleukodystrophy; commercial execution; manufacturing investment

VectorY Strikes $1.2B Option and License Pact with Shape Therapeutics for Brain-Penetrant Gene Therapy Delivery

3 months ago talkbio0Tagged Alzheimer 's disease, Antibodies, biobucks, brain-penetrant delivery, Capsid, Dependovirus, gene therapy, Huntington Disease, milestone payments, Neurodegenerative Disorders, pipeline, SHP-DB1, vectorized, VectorY

VectorY Therapeutics; Shape Therapeutics; SHP-DB1; adeno-associated virus (AAV) capsid; brain-penetrant delivery; gene therapy; neurodegenerative diseases; Huntington’s disease; Alzheimer’s disease; biobucks; pipeline; milestone payments; vectorized antibodies

Capsida Suspends Gene Therapy Trial After First Patient Death

3 months ago talkbio0Tagged CAP-002, Capsida Biotherapeutics, Clinical Trial Suspension, Encephalopathies, gene therapy, Patient Death, STXBP1 gene, United States Food and Drug Administration

Capsida Biotherapeutics; gene therapy; CAP-002; clinical trial suspension; STXBP1 encephalopathy; patient death; FDA

FDA Launches New Submission Program for Rare Disease Gene Therapies via CDER and CBER

3 months ago talkbio0Tagged Accelerated, Approved, CBER, CDER, gene therapy, Hereditary Diseases, Infrequent, RDEP, regulatory guidance, submission program, United States Food and Drug Administration

CDER; CBER; rare disease; gene therapy; FDA; submission program; accelerated approval; RDEP; regulatory guidance; rare genetic disorders

Recent News on Scaling Up Cell & Gene Therapy Supply Chains (2025)

3 months ago talkbio0Tagged Automation, Cells, digital solutions, gene therapy, Growth, Logistics, Market, patient-specific therapies, risk mitigation, scalability, supply chain, white glove delivery

cell and gene therapy; supply chain; scalability; logistics; digital solutions; risk mitigation; white glove delivery; patient-specific therapies; automation; market growth

Editas Medicine Reboots With Cholesterol Gene Therapy (EDIT-401) as Lead Asset

3 months ago talkbio0Tagged Clinical Development, EDIT-401, Editas Medicine, Gene Modification, gene therapy, Hyperlipidemia, in vivo, LDL Cholesterol Lipoproteins, LDLR gene, strategic pivot

Editas Medicine; EDIT-401; gene therapy; hyperlipidaemia; LDL cholesterol; LDLR gene; in vivo gene editing; strategic pivot; clinical development

Vertex forges $2B-plus alliance with Enlaza to create new autoimmune drugs, improve conditioning methods

3 months ago talkbio0Tagged Autoimmune Diseases, conditioning regimens, covalent biologics, drug conjugates, engagers, Enlaza, gene therapy, T-Lymphocyte, War-Lock platform

Vertex Pharmaceuticals; Enlaza Therapeutics; autoimmune diseases; gene therapy; conditioning regimens; War-Lock platform; covalent biologics; drug conjugates; T-cell engagers; biotech alliance

Vinay Prasad Returns to the FDA With Fewer Titles and a Lower Profile

4 months ago talkbio0Tagged Center for Biologics Evaluation and Research, gene therapy, Lower Profile, Prasad, research, Return to (contextual qualifier) (qualifier value), Titles, United States Food and Drug Administration, vaccine regulation, Vinay

Vinay Prasad; FDA; return; Center for Biologics Evaluation and Research (CBER); fewer titles; lower profile; gene therapy; vaccine regulation

FDA Lifts Hold on Rocket Pharmaceuticals’ Pivotal Gene Therapy Study at Lower Dose

4 months ago talkbio0Tagged C3 complement inhibitor, Capillary Leak Syndrome, Dosage, gene therapy, Glycogen Storage Disease Type IIb, Individual Adjustment, Phase 2 Clinical Trials, Rocket, RP-A501, Study on Hold, United States Food and Drug Administration

FDA; Rocket Pharmaceuticals; gene therapy; Danon disease; clinical hold; RP-A501; dose adjustment; C3 complement inhibitor; capillary leak syndrome; patient death; Phase II trial