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.
Researchers have identified KpsM as a virulence factor in Escherichia coli that was responsible for liver damage in alcohol-associated hepatitis (AH). A small-molecule inhibitor of KpsM reduced liver damage in animal models of AH.
Biopharma happenings, including deals and partnerships, grants, preclinical data and other news in brief: Altamira, Ayrmid, Bioinvent, Biolinerx, Boundless Bio, Galmed, Hansa, Novartis, Rheacell, Regulus, Sanofi, Xoma.
Clinical updates, including trial initiations, enrollment status and data readouts and publications: Asklepios, Bayer, Bioxcel, Gilgamesh, Immutep, Innovent, Invivyd, Iolyx, Mineralys, Palisade, Rocket.
The U.S. Trade Representative (USTR) is asking for help in its search for “freeloaders” that refuse to shoulder their share of the cost of biopharma R&D.
Glycoengineering specialist Glycoera AG is preparing to take its extracellular protein degrader constructs into the clinic in the treatment of autoimmune diseases, after closing a $130 million series B.
Epimab Biotherapeutics Inc. licensed out a development-ready KLK2/CD3 bispecific T-cell engager (TCE) for advanced prostate cancer to Juri Biosciences Inc. through a potential $210 million deal.
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