After seeing improved muscle function in phase 1/2 trial, Regenxbio launches pivotal DMD study
Regenxbio’s investigational gene therapy improved muscle function for patients with Duchenne muscular dystrophy (DMD) in a phase 1/2 study. After discussions with the FDA, the biotech has launched a pivotal study and plans on filing for accelerated approval in 2026....
Intellia Announces First Clinical Evidence from Ongoing Phase 1 Study that Nexiguran Ziclumeran (nex-z), an In Vivo CRISPR/Cas9-Based Gene Editing Therapy, May Favorably Impact Disease Progression in Transthyretin (ATTR) Amyloidosis
Consistently rapid, deep and durable reduction in serum TTR accompanied by evidence of disease stabilization or improvement after a one-time treatment of nex-z, supporting the hypothesis that greater TTR reduction may lead to a greater clinical benefit in ATTR...
[2024/11/15] Gene and Cell Therapy- weekly digest from PackGene
FeaturedNewsArticlesPackGene's NewsletterReceive the latest news and insights to your inbox.About PackGenePackGene Biotech is a world-leading CRO and CDMO, excelling in AAV vectors, mRNA, plasmid DNA, and lentiviral vector solutions. Our comprehensive offerings span...
One-time gene therapy reverses liver disease MASH in mouse models
Credit: Molecular Therapy (2024). DOI: 10.1016/j.ymthe.2024.10.023UAB researchers have reversed metabolic dysfunction-associated steatohepatitis (MASH) in mouse models. MASH is a severe liver disease associated with obesity and type 2 diabetes that affects more than...
PTC Therapeutics Achieves FDA Approval for First Brain-Administered Gene Therapy
Breakthrough Approval for Kebilidi The FDA has granted a landmark approval to PTC Therapeutics' Kebilidi, making it the first gene therapy in the U.S. to be administered directly into the brain. This approval represents a major step forward in gene therapy, offering...
Big Pharma’s Next Move: BMS, Merck, Amgen Eye M&A to Tackle Patent Expirations
Top drugmakers face a wave of patent expirations, pressuring them to acquire new products for near-term launches. A recent report highlights the looming loss of exclusivity (LOE) for major pharma firms between 2025-2030 and 2030-2040. BMS, Merck, Amgen, Novartis, and...
FDA Approves Autolus’ New CAR-T Therapy for Aggressive Blood Cancer
In a significant development for cancer treatment, the US Food and Drug Administration (FDA) has approved Autolus Therapeutics’ CAR-T cell therapy for adult patients with relapsed or treatment-resistant B cell precursor acute lymphoblastic leukemia (B-ALL). This...
Inflammation-reducing molecule provides a new target for treating atherosclerosis
Systemic metrics of atherosclerosis resolution in dietary cessation CPC model. Credit: Cell Reports (2024). DOI: 10.1016/j.celrep.2024.114911Researchers at Case Western Reserve University have identified a new target to treat atherosclerosis, a condition where plaque...
Ireland’s SSPC and NIBRT Team to Take on Biopharma Bottlenecks
The Research Ireland Centre for Pharmaceuticals (SSPC) has partnered with Ireland’s National Institute for Bioprocessing Research and Training (NIBRT) to develop technologies to address R&D and manufacturing challenges facing the sector. The manufacturability of...
Continuity Biosciences Launches to Enable Breakthrough Therapies with Innovative Delivery Technologies
Continuity Biosciences, LLC, a new bioscience company dedicated to developing and commercializing cutting-edge technologies for cell reprogramming, immune modulation, and drug delivery, is excited to announce its official launch. The company seeks to bridge...
PTC Therapeutics Announces FDA Approval of AADC Deficiency Gene Therapy
- First-ever FDA approval for gene therapy directly administered to the brain - - Priority review voucher granted - - Broad label including children and adults - - PTC pioneers new approach for CNS drug delivery - WARREN, N.J., Nov. 13, 2024 /PRNewswire/ -- PTC...
Trump picks vaccine skeptic Kennedy to lead HHS, in what would be an upheaval of US health policy
President-elect Donald Trump has picked Robert F. Kennedy Jr. to be his next HHS secretary, likely giving the government’s most powerful healthcare post to a vaccine skeptic whose controversial views would be a huge break with decades of US health and scientific...
Popular Services
AAV Packaging Service
AAV Analytical Service
Vector Design