Denali Therapeutics Inc.’s extensive update on clinical programs in central nervous system diseases at the start of this year included plans for lead asset DNL-310 in mucopolysaccharidosis II (MPS II), also known as Hunter syndrome – a space where other notable players include such names as Regenxbio Inc. and Takeda Pharmaceutical Co. Ltd.
Denali Therapeutics Inc.’s extensive update on clinical programs in central nervous system diseases at the start of this year included plans for lead asset DNL-310 in mucopolysaccharidosis II (MPS II), also known as Hunter syndrome – a space where other notable players include such names as Regenxbio Inc. and Takeda Pharmaceutical Co. Ltd.
Researchers from Children's Hospital Los Angeles presented data from a study that linked a homozygous missense mutation in ARSK to a new subtype of the lysosomal storage disease mucopolysaccharidoses (MPS).
The FDA has cleared Immusoft Corp.'s IND for ISP-001 (iduronicrin genleukocel-T) for the treatment of mucopolysaccharidosis type I (MPS I), allowing initiation of a phase I trial.
In its first big pharma deal since it was founded around a cell programming technology in 2009, Immusoft Corp. signed Takeda Pharmaceutical Co. Ltd. to a research collaboration and license option targeting rare inherited metabolic disorders. The agreement brings an undisclosed up-front fee and research funding to Immusoft, which is also eligible to earn more than $900 million if all options are exercised and all milestones hit.
In its first big pharma deal since it was founded around a cell programming technology in 2009, Immusoft Corp. signed Takeda Pharmaceutical Co. Ltd. to a research collaboration and license option targeting rare inherited metabolic disorders. The agreement brings an undisclosed up-front fee and research funding to Immusoft, which is also eligible to earn more than $900 million if all options are exercised and all milestones hit.
One of 19 children dosed with an experimental gene therapy for Sanfilippo syndrome has died following the treatment, given as part of a phase II/III trial of the medicine run by its developer, Paris-based Lysogene SA. The trial has been on clinical hold since June, but the immediate cause of the death is unknown, the company said.
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).
Ultragenyx Pharmaceutical Inc.’s top-line win in January with DTX-301 gene therapy in ornithine transcarbamylase (OTC) deficiency seemed to presage even better things to come later this year, and analysts more recently hailed fourth-quarter earnings that showed satisfying progress with Crysvita (burosumab).
