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.
Barely a year after the U.S. FDA shackled Abeona Therapeutics Inc.’s cell-based gene therapy with a complete response letter, the agency has approved it for treating a rare and genetic skin disease. Zevaskyn (prademagene zamikeracel), for treating wounds in adult and pediatric patients with recessive dystrophic epidermolysis bullosa, will be priced in the U.S. at $3.1 million.
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.
Positive early stage data for Verve Therapeutics Inc.’s base editing therapy points to a range of development options, including bringing partner Eli Lilly and Co. in a little closer. The new data helped ease the company’s pain from the April 2 enrollment pause of a similarly designed therapy from Verve. Verve’s Heart-2 phase Ib of VERVE-102 in treating 14 patients with heterozygous familial hypercholesterolemia and/or premature coronary artery disease showed one infusion led to dose-dependent decreases in blood PCSK9 protein levels and low density lipoprotein cholesterol.
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.
Genflow Biosciences plc has announced a development program in ophthalmology focused on advancing a novel gene therapy leveraging its proprietary centenarian SIRT6 (cSIRT6).
Researchers from Voyager Therapeutics Inc. presented preclinical activity data of VY-1706, a blood-brain barrier (BBB)-penetrant gene therapy comprising an adeno-associated virus serotype 9 capsid (AAV9-C9P39) vector encoding primary artificial microRNA (pri-amiRNA) consisting of short-interfering RNA (siRNA) targeting human microtubule-associated protein tau (MAPT) protein.
Metachromatic leukodystrophy (MLD) is an autosomal recessive lysosomal storage disease originating from biallelic pathogenic variants in the ARSA gene, mainly affecting young children.
RSS
