EG 427 SAS has received IND clearance from the FDA for EG-110A, a gene therapy for the treatment of neurogenic detrusor overactivity in patients with spinal cord injury. A phase Ib/IIa study is being initiated.
Astellas Pharma US Inc., a U.S. affiliate of Astellas Pharma Inc., has entered into a sponsored research agreement with the University of Massachusetts Medical School (UMass Chan Medical School) to conduct research for an AAV vector-mediated gene therapy for the treatment of Alexander disease.
Opus Genetics Inc. has received $1.7 million in project-based funding from the Foundation Fighting Blindness to help advance two preclinical candidate programs for inherited retinal diseases.
The FDA has granted orphan drug designation to Be Biopharma Inc.’s BE-101, a novel engineered B-cell medicine being developed for the treatment of hemophilia B.
Researchers from Purespring Therapeutics Ltd. and affiliated organizations have presented preclinical data for the adeno-associated vector (AAV) gene therapy PS-001 for the treatment of glomerular disease.
Alpha-1 antitrypsin deficiency (AATD) is a recessive genetic disorder caused by single nucleotide variants (SNV) in the SERPINA1 gene encoding for alpha-1 antitrypsin (AAT), with the most common mutation being the E342K mutation (Z allele) that introduces an amino acid change from glutamic acid (E) coding for M-AAT to a lysine (K) coding for Z-AAT.
Avirmax Biopharma Inc. has received IND approval from the FDA to initiate a phase I/IIa trial for its gene therapy treatment targeting wet age-related macular degeneration (AMD), including polypoidal choroidal vasculopathy (PCV).
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