Amarna Therapeutics BV is advancing Nimvec AM-510 gene therapy for type 1 diabetes and has formed a scientific advisory board to guide development.

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).

Kate Therapeutics Inc. recently presented data on a novel muscle- and heart-targeted, liver de-targeted development candidate for the treatment of Duchenne muscular dystrophy (DMD) – KT-809.

Precision Biosciences Inc. has received pre-IND regulatory feedback from the FDA and ex-U.S. agencies providing alignment and clarity on Precision's final IND/CTA-enabling preclinical plans and clinical strategy for PBGENE-HBV.

Uniqure NV has received FDA clearance of its IND application for AMT-260, the company’s gene therapy candidate that represents a potential one-time administered approach to treating refractory mesial temporal lobe epilepsy (MTLE).