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.
As many had predicted following news of a clinical hold on the gene therapy last month, Regenxbio Inc. disclosed receipt of a complete response letter (CRL) regarding its BLA for RGX-121 (clemidsogene lanparvovec) in mucopolysaccharidosis II, an ultrarare neurodegenerative disease in dire need of new therapies.
Laminins are extracellular matrix (ECM) glycoproteins that preserve the structural and functional integrity of tissues. These heterotrimeric proteins, composed of one α, β and γ chain encoded by different genes, provide mechanical support, facilitate cell adhesion and maintain tissue organization and stability.
Affinia Therapeutics Inc. has obtained IND clearance from the FDA for AFTX-201, an investigational genetic medicine for the treatment of BAG3-associated dilated cardiomyopathy (DCM). The phase I/II UPBEAT trial will begin in the first half of this year.
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