Opus Genetics Inc. has obtained IND clearance from the U.S. FDA application for OPGx-BEST1, a gene therapy for the treatment of bestrophin-1 (BEST1)-related inherited retinal disease (IRD).

The U.S. FDA has given a swift and full approval to Precigen Inc.’s gene therapy, Papzimeos (zopapogene imadenovec), for treating adults with recurrent respiratory papillomatosis (RRP), a rare and chronic disease characterized by benign tumors in the respiratory tract.

Aussie gene therapy company Mirugen Pty Ltd. has emerged from stealth mode from the Center for Eye Research Australia in Melbourne, toting a AU$4.5 million (US$2.9 million) seed round that will see it optimize its lead candidate to treat retinitis pigmentosa.

Childhood-onset neurodegeneration with cerebellar atrophy (CONDCA) is an autosomal recessive disorder that causes progressive motor and cognitive impairment in children. The disease arises as a result of inactivating mutations in cytosolic carboxypeptidase 1 (CCP1), leading to excessive polyglutamylation of tubulin in the brain. Researchers at Shimane University have shown in a mouse model that delivering a truncated form of CCP1 into the brain can substantially mitigate Purkinje cell degeneration and improve motor function.

CLN3 disease, also known as juvenile neuronal ceroid lipofuscinosis, is a rare neurodegenerative disorder affecting neuronopathic lysosomal storage that severely impacts the central nervous system while also inducing notable peripheral neuromuscular symptoms. Researchers from Washington University School of Medicine have demonstrated the potential of gene therapy for CLN3 disease.

Precision medicine is becoming a reality in Asia Pacific as more targeted therapies are being developed that are tailored to individual patients, offering a potential cure for disease. But is Asia Pacific ready to harness this transformation, and if not, what are the hurdles that need to be cleared?

After about a two-week absence as the U.S. FDA’s CBER director, Vinay Prasad’s return overall prompted a mild reaction on Wall Street for some stocks tied to companies developing cell and gene therapies.

Precision medicine is becoming a reality in Asia Pacific as more targeted therapies are being developed that are tailored to individual patients, offering a potential cure for disease. But is Asia Pacific ready to harness this transformation, and if not, what are the hurdles that need to be cleared? A new report by LEK Consulting examines Asia Pacific’s readiness for what it calls the “Precision Era,” and examines four key biopharma markets in Australia, China, Japan and South Korea.

Purespring Therapeutics Ltd. has received the go-ahead for a phase I/II trial of its investigational gene therapy PS-002 from both the U.K. Medicines and Healthcare products Regulatory Agency (MHRA) and the National Health Service Health Research Authority and Research Ethics Committee.

In an effort to develop next-generation treatments for cystinuria, researchers at Vanderbilt University and Tennessee Valley Health Services have used a non-viral piggyBac transposon approach to insert the Slc3a1 gene into one kidney of mice lacking the endogenous gene, which services as a model of type A cystinuria.