Duchenne muscular dystrophy

Jun 09, 2023

The cell and gene therapy market has seen a revolutionary transition in recent years, with advancements in scientific research and novel methods of treatment driving a rise in development activities. This has resulted in an increase in the number of cell and gene therapy choices available to patients suffering from...

May 16, 2023

Sarepta Therapeutics Announces Positive Vote from U.S. FDA Advisory Committee Meeting for SRP-9001 Gene Therapy Sarepta Therapeutics, Inc., a pioneer in precision genetic medicine for rare diseases, announced that the FDA's Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) voted 8 to 6 in favor of...

Nov 01, 2022

Actinium Announces Positive Top-line Results from Pivotal Phase III SIERRA Trial of Iomab-B Actinium Pharma is on track to submit its targeted radiotherapy for AML patients requiring a bone marrow transplant in the United States, boosted by top-line data from a pivotal trial. The SIERRA trial of Iomab-B, an anti...

Oct 04, 2022

Zealand Pharma Announces the Positive Topline Results from its Phase III trial of Glepaglutide Zealand Pharma A/S, a biotech company specializing in peptide-based medicines, announced positive topline results from its phase III trial of glepaglutide. In the evenly randomized double-blind trial, 106 patients with...

Aug 02, 2022

Bristol-Myers Squibb’s Opdivo and Yervoy Combo Fails in Phase III Trial Bristol-Myers Squibb has reported that their Opdivo and Yervoy checkpoint inhibitor combo failed a phase III trial as adjuvant (post-surgery) therapy for renal cell carcinoma (RCC), the most frequent type of kidney cancer. The CheckMate -914...

Nov 01, 2021

Progress is driven by innovation. When it comes to developing novel medications and therapeutic biological products, the FDA's Center for Drug Evaluation and Research (CDER) assists the pharmaceutical sector at every stage of the process. CDER offers scientific and regulatory assistance needed to bring innovative m...

Oct 05, 2021

Pfizer tightens DMD patient criteria after serious adverse events in phase 3 gene therapy trial Pfizer is tightening up the criteria for Duchenne muscular dystrophy patients to partake in phase 3 clinical trials of its experimental gene therapy after three severe events were cropping up in ongoing studies. Th...

Dec 17, 2019

Wave Life Sciences has recently announced the discontinuation of its drug suvodirsen, for the treatment of a rare Duchenne Muscular Dystrophy mutation.  Duchenne Muscular Dystrophy is a progressive worsening of the skeletal and heart muscles resulting in weakness. The disorder, is due to deficient prot...

Dec 05, 2019

Bristol-Meyers Squibb and Acceleron Pharma have announced that US FDA committee will review BMS’s supplemental Biologics License Application (sBLA) for its Reblozyl in MDS. Reblozyl ((luspatercept-aamt) has recently been approved by the regulatory authority to cure a rare type of blood disorder, Beta-thalassemi...

Aug 20, 2019

Juvenescence closes a USD 100 Million Series B round Juvenescence, a life sciences company focused on treating ageing problems, has successfully raised USD 100 Million in a Series B round. The investors who took part in the funding were Grok Ventures, the investment company of Mike Cannon-Brookes (Atlassian co-...