How the U.S. FDA might respond became a serious question for Wall Street as Sarepta Therapeutics Inc. made known a second death due to acute liver failure with gene therapy Elevidys (delandistrogene moxeparvovec), cleared for Duchenne muscular dystrophy (DMD). Shares of Cambridge, Mass.-based Sarepta (NASDAQ:SRPT) closed June 16 at $20.94, down $15.24, or 42%, as Wall Street digested the news.
Researchers at Ileadbms Co. Ltd. have announced promising results of their novel drug IL-21120033 in a preclinical model of rheumatoid arthritis. The drug acts as a selective agonist of the chemokine receptor CXCR7.
Regenxbio Inc.’s gene therapy in treating Duchenne muscular dystrophy (DMD) produced positive initial phase I/II results from its first five patients. However, the company’s stock (NASDAQ:RGNX) shuddered on June 5 as shares closed at $8.36 each, a drop of 17% on the day.
Inflamax Pharmaceuticals (Guangzhou) Co. Ltd. has disclosed proprotein convertase subtilisin/kexin-type 6 (PCSK6; PACE4; PC7A) inhibitors reported to be useful for the treatment of osteoarthritis.
Developing a therapy for an ultra-rare condition has its challenges, including finding enough patients for clinical enrollment and convincing regulatory authorities that limited data prove the candidate is safe and effective. For that reason, Stealth Biotherapeutics Inc. has faced numerous roadblocks getting its mitochondria-targeting elamipretide across the finish line for Barth syndrome, a condition that affects about 230 to 250 males worldwide, including fewer than 150 in the U.S.
Inclusion body myositis (IBM) is the most common acquired myopathy in people over 50 years of age, characterized by chronic and progressive muscle weakness, where its pathogenesis involves inflammatory and degenerative pathways that are not well understood to date.
Annji Pharmaceutical Co. Ltd.’s rosolutamide (AJ-201, JM-17) achieved positive results in a phase I/II trial in adults with spinal and bulbar muscular atrophy, also known as Kennedy’s disease.
Alteogen Inc. completed a merger between two subsidiaries – Altos Biologics Inc. and Alteogen Healthcare Inc. – branding the new entity as Alteogen Biologics Inc.
Alteogen Inc. completed a merger between two subsidiaries – Altos Biologics Inc. and Alteogen Healthcare Inc. – branding the new entity as Alteogen Biologics Inc.
Rheumatoid arthritis, atopic dermatitis, ulcerative colitis and several other inflammatory diseases involve hyperactivation of JAK-STAT signaling, in which the kinase JAK binds and phosphorylates the transcription factor STAT, which then turns on gene expression.
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