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).

Previous research has demonstrated that activin receptor-like kinase 7 (ALK7) signaling suppresses lipolysis, resulting in increased adipocyte size and lipid content. Additionally, predicted loss of function variants in the ALK7 gene (ACVR1C) have been associated with reduced waist-to-hip ratio (WHR) adjusted for body mass index, protection from type 2 diabetes, as well as reduced risk of cardiovascular disease.