News

Idera Details Working of 3G Antisense Technology, Potential FSHD Therapy

Idera Pharmaceuticals recently presented pre-clinical data regarding the gene-silencing mechanisms of its third generation antisense (3GA) technology platform, a potential therapeutic for diseases that include, but are not limited to, facioscapulohumeral muscular dystrophy (FSHD). The presentation, “Precise excision of targeted RNA by third generation antisense (3GA) oligonucleotides” was made at the…

Potential DMD Therapy, Ezutromid, Shows Effectiveness in New Formulation

Summit Therapeutics reports that a new formulation of ezutromid (called F6), its Duchenne muscular dystrophy (DMD) treatment candidate, achieved a greater than six-fold increase in maximum plasma levels in DMD patients participating in a Phase 1 clinical trial, compared to results achieved with its current formulation (F3), and at a lower…

Tivorsan’s Biglycan Granted Orphan Drug Designation for Treatment of Duchenne MD

Tivorsan’s lead investigational therapy for the treatment of Duchenne muscular dystrophy (DMD) has been granted Orphan Drug Designation by the U.S. Food and Drug Administration (U.S. FDA). The human recombinant Biglycan (TVN-102), a natural component of muscles, activates a utrophin-based compensatory pathway that can potentially overcome damage caused by the…

FDA Grants Priority Review to Marathon’s Deflazacort for Duchenne Muscular Dystrophy

New Drug Applications (NDA) for Marathon’s investigational drug deflazacort have been accepted for filing and granted Priority Review by the U.S. Food and Drug Administration (FDA). The NDAs, one for immediate-release tablet formulations and one for an oral suspension formulation, request that deflazacort be approved for the treatment of Duchenne muscular dystrophy…

PhaseBio Doses First Patients in Two-Part Phase 2a Trial for Cardiopulmonary Disorder Drug

The first patients have been dosed in PhaseBio Pharmaceuticals‘ part one of a Phase 2a clinical trial for PB1046, a once-weekly vasoactive intestinal peptide (VIP) receptor activator currently in development to treat cardiopulmonary disorders. Part one is designed to assess the effect of PB1046 in adult patients with stable heart failure with reduced ejection fraction…

Pfizer Acquires Bamboo Therapeutics, Readies Clinical Trial of Gene Therapy for DMD

Bamboo Therapeutics has been fully acquired by Pfizer, considerably expanding that company’s holdings in gene therapy. With the acquisition, Pfizer gains several potential therapeutic agents for rare diseases, including one for Duchenne muscular dystrophy (DMD), soon to enter clinical testing; and one Phase 1/2 clinical stage agent, a potential best-in-class recombinant Adeno-Associated Virus (rAAV)…