Engene Holdings Inc.’s protocol amendment to its phase II trial with detalimogene voraplasmid in bladder cancer worked out in a big way, and shares of the firm closed Nov. 11 at $8.82, up $2.81, or 47%. Engene rolled out additional preliminary data from the pivotal cohort of the ongoing Legend study testing the nonviral gene therapy in high-risk, Bacillus Calmette-Guérin-unresponsive non-muscle invasive bladder cancer with carcinoma in situ, with or without concomitant papillary disease.
In a new publication in Molecular Therapy, researchers from Paracelsus Medical University, Salzburg, Austria, and collaborators present a promising prime editing strategy for junctional epidermolysis bullosa (JEB) treatment.
The U.K. Medicines and Healthcare products Agency (MHRA) has committed to major reforms of how it regulates drugs for rare diseases, making it easier to run clinical trials and get approvals. The new rules will be published in full early in 2026 and come into effect later in the year, but following consultation with industry, academics and patients’ groups, the agency has released a position paper setting out its plans.
Biomarin Pharmaceutical Inc. plans to divest its pioneering gene therapy for hemophilia, Roctavian (valoctocogene roxaparvovec), and remove what had been considered a potential blockbuster from the portfolio in order to grow the company.
Diabetic kidney disease (DKD) is the most prevalent long-term complication of diabetes, affecting up to 30% of individuals with type 1 diabetes within the first 10 years of diagnosis.
Alpha-1 antitrypsin deficiency (AATD) is a monogenic disease caused by mutations in the SERPINA1 gene, which encodes alpha-1 antitrypsin (AAT), a serine protease inhibitor mainly produced by hepatocytes.
Judo Bio Inc. has announced preclinical data demonstrating that its megalin-STRIKER oligonucleotide therapeutics achieved robust and sustained, kidney cell-selective gene silencing in rodents and nonhuman primates (NHPs).
The full rights to Taysha Gene Therapies Inc.’s lead gene therapy candidate are coming back to the company. In 2022, Astellas Pharma Inc. invested $50 million in Taysha for 15% of the company and the exclusive option to license TSHA-102 for treating the rare disease Rett syndrome. That option has expired, according to Taysha.
The U.S. FDA named the first nine recipients of the recently unveiled commissioner’s national priority voucher (CNPV) program aimed at addressing unmet public health needs by shortening regulatory review times to as little as one to two months. For one of those firms, Disc Medicine Inc., which submitted an NDA for bitopertin for rare genetic disorder erythropoietic protoporphyria in September, that could mean a potential approval before the end of 2025.
Some 1 million people around the world suffer severe neurological problems, such as epilepsy, motor impairment and cognitive dysfunction, because they express insufficient SynGAP1, a GTPase-activating protein that operates postsynaptically. Current therapies can mitigate symptoms but not cure the underlying disease. Researchers at the Allen Institute and collaborators have demonstrated a potential genetic cure for SynGAP1 deficiency.
RSS
