Gene-Therapy Treatments for Tay-Sachs, Sickle Cell to Be Featured in Online Gathering – Barron’s

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Cutting-edge research on rewriting the genes responsible for Tay-Sachs disease, sickle-cell anemia, and other disorders will be presented at this weeks online annual meeting of the American Society for Gene and Cell Therapy. Originally planned as a Boston gathering, the scientific groups get-together became a virtual one because of the Covid-19 pandemic.

From Tuesday through Friday, academic researchers will be presenting their latest data online, along with updates from firms like Bluebird Bio (ticker: BLUE), Voyager Therapeutics (VYGR), Fate Therapeutics (FATE), Beam Therapeutics (BEAM), Axovant Gene Therapies (AXGT), and many others. Patients and their families have found their way to clinical trials through the societys website.

Bluebird plans presentations on its cell therapy against the blood cancer known as multiple myeloma. Using a technology known as CAR-T, the company creates supercharged versions of a patients immune cells that have halted disease progression in some of the 18 patients enrolled in a continuing Phase 1 trial.

Featured on Friday will be reports on the first babies treated with gene therapy for the debilitating neurodegenerative disorder Tay-Sachs. The treatment is being developed by the London-based Axovant under license from the University of Massachusetts Medical School.

Voyager will discuss its preclinical mouse studies on treating neurological disorders like amyotrophic lateral sclerosis and Huntingtons disease by using techniques that block the rogue signals generated by defective genes.

Fate Therapuetics is scheduled to show a new off-the-shelf CAR-T technology that it hopes will allow the immune system to target a broad range of solid tumors as well as multiple myeloma. The approach is licensed from Harvard Universitys Dana-Farber Cancer Institute.

Beam, meanwhile, will detail success it has shown in preclinical editing of the genetic defect that causes sickle-cell anemia. The company is developing a sharper-edged way of rewriting faulty genes than the widely used Crispr technology that Beam licensed from researchers at the Broad institute of Harvard and MIT. Beam founder and Crispr pioneer Feng Zhang will give a featured lecture as part of the online meeting on Thursday.

Write to Bill Alpert at william.alpert@barrons.com

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Gene-Therapy Treatments for Tay-Sachs, Sickle Cell to Be Featured in Online Gathering - Barron's