Apellis Pharmaceuticals Inc. could receive up to $1.25 billion from Swedish Orphan Biovitrum (Sobi) AB in their collaboration to develop systemic pegcetacoplan, a C3 therapy for treating several rare diseases in hematology, nephrology and neurology.
BEIJING – Canbridge Pharmaceuticals Inc. last week won marketing approval in China for its first rare disease drug, the mucopolysaccharidosis II therapy Hunterase (idursulfase beta injection).
BEIJING – Canbridge Pharmaceuticals Inc. last week won marketing approval in China for its first rare disease drug, the mucopolysaccharidosis II therapy Hunterase (idursulfase beta injection).
Millendo Therapeutics Inc. in April decided to scrap work altogether with livoletide in Prader-Willi syndrome after a phase IIb study fell short of statistical significance, but hope remains alive for – among others in the space – Soleno Therapeutics Inc., another recent newsmaker with once-daily diazoxide choline controlled-release tablets in PWS.
DUBLIN – Ipsen SA is on track for an NDA filing for palovarotene in fibrodysplasia ossificans progressiva (FOP), an ultra-rare disease characterized by the gradual replacement of skeletal muscle and connective tissue with bone, following an interim analysis of phase III data which indicates that the drug may have a substantial effect on the disease process.
A third child with X-linked myotubular myopathy (XLMTM) has died after receiving the higher of two doses of an experimental gene therapy for the rare disease under development at Audentes Therapeutics Inc. Preliminary findings indicate that the immediate cause of death was gastrointestinal bleeding, Audentes said. The trial, testing AT-132 (resamirigene bilparvovec), was already on an FDA clinical hold.
While privately held Levo Therapeutics Inc.’s phase III study of LV-101 (intranasal carbetocin) for treating Prader-Willi syndrome (PWS) failed to meet its primary outcome measurements, the company’s CEO told Bioworld the first secondary endpoint showed a statistical significance that raised her hopes the FDA might approve the selective oxytocin-receptor agonist for the indication.
Carmine Therapeutics Inc. said Tuesday that Takeda Pharmaceutical Co. Ltd. has engaged it in a research collaboration aimed at the discovery, development and commercialization of new nonviral gene therapies for two rare disease targets.
Carmine Therapeutics Inc. said Tuesday that Takeda Pharmaceutical Co. Ltd. has engaged it in a research collaboration aimed at the discovery, development and commercialization of new nonviral gene therapies for two rare disease targets. Terms of the deal included an up-front payment of undisclosed value for Carmine, plus research funding, and more than $900 million in potential milestone payments.
BEIJING – Rare disease specialist Canbridge Pharmaceuticals Inc., of Beijing, said it is collaborating with the Horae Gene Therapy Center at the University of Massachusetts (UMass) Medical School to conduct gene therapy research with a focus on neuromuscular conditions.
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