Spark Therapeutics Inc. has presented a proprietary adeno-associated viral (AAV) vector expressing an artificial miRNA targeting human α-synuclein (α-Syn) mRNA. Accumulation of misfolded and insoluble α-Syn causes neuronal toxicity in preclinical models and has been identified as the underlying cause of synucleinopathies.

Variant SAS and the Rare Ocular Diseases Center at the University of Campania Luigi Vanvitelli (UCLV) have received positive feedback from the EMA for VAR-002, a recombinant AAV vector gene therapy targeting inherited retinal dystrophies linked to CRX mutations.

Epicrispr Biotechnologies Inc. has secured $68 million in the first close of its series B financing to support the clinical development of EPI-321, a first-in-class epigenetic therapy for facioscapulohumeral muscular dystrophy (FSHD).

Hubble Therapeutics LLC has successfully closed a $7.3 million series A funding round to support progression of its lead candidate, HUB-101, into clinical trials.

Bladder cancers (BCs) that invade the muscle layer are classified as muscle-invasive bladder cancers (MIBCs). The MIBC subtype accounts for around 25% of all BC cases, with a significant proportion of patients presenting distant metastases.

Sineugene Therapeutics Co. Ltd. has obtained IND clearance from the FDA for SNUG-01, a first-in-class tripartite motif protein 72 (TRIM72)-targeted gene therapy candidate for amyotrophic lateral sclerosis (ALS). A phase I/IIa trial will evaluate SNUG-01 in adults with ALS.

Investigators from Insmed Inc. have presented new preclinical data on the efficacy of their adenoviral vector (AAV9)-based gene therapy INS-1201 for the treatment of Duchenne muscular dystrophy (DMD).

Bristol Myers Squibb Co.’s decision this week to snag Bluebird Bio Inc. spinout 2seventy Bio Inc. for $102 million net – just weeks after investors bid $30 million for Bluebird itself – seemed to place a final blow on what was once a promising gene therapy company. The space in general has struggled to make business sense out of the one-time therapies that often involve complicated manufacturing and exorbitant prices, despite the life-changing value that gene therapies bring to patients. But despite some recent setbacks, biopharmas continue to plow forward with promising research in the field.

Pathogenic variants in the GJB2 gene are the most common genetic cause of congenital sensorineural hearing loss and are mostly associated with an autosomal recessive non-syndromic deafness 1A (DFNB1A).