Capsida Biotherapeutics Inc. has gained IND clearance from the FDA for CAP-002, its first-in-class, intravenously administered gene therapy for syntaxin-binding protein 1 developmental and epileptic encephalopathy (STXBP1-DEE). Dosing in the phase I/IIa SYNRGY trial will begin in the third quarter of this year.
Genprex Inc. and University of Pittsburgh have entered into a new sponsored research agreement (SRA) to study GPX-002, Genprex’s gene therapy for diabetes in animal models of type 1 and type 2 diabetes.
“I’m a pediatrician in metabolic diseases, and every day in my clinical work I’m confronted with our lack in effective therapies for our patients.” That was the sobering introduction by Sabine Fuchs in her talk at the 2025 Congress of the European Association for the Study of the Liver in Amsterdam this week. The nature of metabolic diseases makes it difficult to develop treatments for them. “There are over 1,500 diseases known by now, and it is just very difficult to develop therapies for each and every individual rare disease.”
Nuevocor Pte. Ltd. has closed a $45 million series B, enabling it to move lead gene therapy NVC-001 into the clinic in the treatment of an inherited form of cardiomyopathy.
Odylia Therapeutics Inc. has announced it is working under a codevelopment partnership with the NPHP1 Family Foundation to create an AAV-based gene replacement therapy for retinal dystrophy caused by mutations in the NPHP1 (nephrocystin-1) gene.
Mammoth Biosciences Inc. has nominated its first clinical development candidate – MB-111 – a potential one-time treatment for patients with very high triglycerides, including familial chylomicronemia syndrome (FCS) and severe hypertriglyceridemia. IND-enabling studies are on track to begin this year.
Blackfinbio Ltd. has obtained IND clearance from the FDA for its novel AAV gene therapy, BFB-101, for hereditary spastic paraplegia type 47 (SPG47), which is caused by changes in the AP4B1 gene. A phase I/II trial will be conducted in the U.S. at Boston Children’s Hospital.
Dravet syndrome (DS) is a rare and severe form of epilepsy that causes intellectual disability and motor deficits and can lead to premature death. A loss-of-function mutation in one copy of SCN1A, encoding the voltage-gated sodium channel (NaV)1.1 α subunit, is the most frequent alteration found in DS patients and has been linked with inhibitory neuron dysfunction. Despite the potential of gene therapies, AAV-mediated SCN1A gene replacement for DS has not been possible yet due to AAV genome size constraints.
Despite being known for more than 150 years, Duchenne muscular dystrophy (DMD) remains an untreatable disease affecting approximately 1 of every 3,500-5,000 males. Muscles in patients express no or inactive dystrophin, rendering them weak and less mobile.
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