central nervous system disorder
JCR Pharmaceuticals, Modalis Therapeutics Ink Research Deal to Develop CNS Gene Therapy
The gene therapy for an undisclosed central nervous system disease is based on JCR's delivery technology and Modalis' epigenome editing platform.
4D Molecular Therapeutics, Arbor Biotechnologies Partner on Nerve Disorder Gene Therapies
The companies will trade taking lead on developing and commercializing up to six therapeutic candidates for rare and common central nervous system (CNS) diseases.
Taysha Gene Therapies Expecting $150M from PIPE Financing; Provides Pipeline Updates
The firm will use the PIPE financing and its available cash to advance gene therapies for rare central nervous system disorders and fund operations into Q3 2025.
Scipher Medicine, Ionis Pharmaceuticals Collaborate on Oligonucleotide Precision Drugs
The deal allows the companies to leverage data from Scipher's Spectra platform and identify subgroups of patients most likely to benefit from treatment.
Ultragenyx to Study Angelman Syndrome Treatment in US With FDA Protocol Amendment Approval
The company can now align doses of the investigational antisense oligonucleotide therapy in pediatric patients enrolled at US and ex-US trial sites.