Tessera Therapeutics Inc.’s lead in vivo gene editing program, TSRA-196, has been awarded orphan drug and fast track designations by the FDA for adults with α-1 antitrypsin deficiency (AATD).
A new study by researchers at Stanford University and collaborating institutions aimed to investigate the safety and efficiency of lipid nanoparticle-mediated Cas13d mRNA delivery to uveal melanoma cells.
Researchers from Harvard Medical School and Spark Therapeutics Inc. tested subretinal NRF2 gene therapy in dry AMD models to investigate whether it could relieve oxidative stress and inflammation.
Nonprofit Finding Hope for Frizzle (FRRS1L) and Apertura Gene Therapy have signed a license agreement for the development of a gene therapy for FRRS1L disease, also known as early infantile epileptic encephalopathy type 37, using Apertura’s CNS-targeting TfR1 CapX AAV capsid.
Researchers from University College London and collaborating institutions have recently published results from their study aiming to optimize gene therapy for CLN5 disease.
Hunter syndrome, also called mucopolysaccharidosis II, is an X-linked genetic lysosomal disorder caused by loss-of-function mutations in the IDS gene, encoding iduronate-2-sulfatase (I2S). I2S is a lysosomal enzyme responsible for the cleavage of glycosaminoglycans (GAGs), and its deficiency results in accumulation of GAGs leading to a multisystemic disorder.
A recent study published in Molecular Therapy by researchers from West China Hospital at Sichuan University and collaborators aimed to investigate the effect of CaMKII gene therapy on retinal ganglion cell (RGC) protection in mouse models of retinal ischemia/reperfusion (I/R) injury and congenital glaucoma.
Friedreich’s ataxia (FA), the most common form of hereditary ataxia, is an autosomal recessive neurodegenerative disorder affecting multiple organ systems, and causing cardiomyopathy, scoliosis, muscle weakness, speech impairment and other systemic issues.
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