The allure of gene therapy was proved yet again as Waltham, Mass.-based Affinia Therapeutics Inc. bagged an oversubscribed $60 million series A financing to boost the push for drugs to benefit people affected by muscle and central nervous system conditions.
With a phase IIb readout coming in the third quarter of 2020, Cambridge, Mass.-based Fulcrum Therapeutics Inc. might be set up for a win in facioscapulohumeral muscular dystrophy (FSHD), a genetic muscle disorder for which there’s no treatment.
Keros Therapeutics Inc. CEO Jasbir Seehra told BioWorld that he plans to use at his new company lessons learned as co-founder of Acceleron Pharma Inc., where work with receptors in the TGF-beta superfamily “taught me the potential of the biology and those molecules, but also the limitations” with regard to safety that need to be surmounted.
Vertex Pharmaceuticals Inc. brought in $4.16 billion in product revenues in 2019, bolstered in large part by the late October approval of Trikafta, earning $420 million in the few weeks that remained in the year.
Expectations that a phase III trial of Ipsen SA's palovarotene will miss its primary endpoint of reducing abnormal bone growth among people with a rare bone disorder led the company to pause dosing in that study and another as it evaluates next steps.
BEIJING – China-U.S. biotech Transcenta Holding Ltd., headquartered in Shanghai and Boston, completed a series B+ financing round to secure $100 million to continue its efforts in developing oncology and bone disorder drugs and to prepare for an IPO.
PERTH, Australia – On the heels of a $1.15 billion deal between Roche Holding AG and Sarepta Therapeutics Inc. for Sarepta’s gene therapy to treat Duchenne muscular dystrophy (DMD), another therapy to treat DMD is emerging from Down Under.
A medicine Stealth Biotherapeutics Inc. has been developing for the potential treatment of primary mitochondrial myopathy (PMM) missed the co-primary endpoints of a key phase III trial, sending company shares (NASDAQ:MITO) down 65.9% to close at $4.75 on Friday.
In August the FDA was skeptical about Sarepta Therapeutics Inc.’s injectable Vyondys 53 (golodirsen), but that changed swiftly Friday with the agency’s accelerated approval for the Duchenne muscular dystrophy (DMD) follow-on therapy, the first treatment specifically for this subtype.