March was a banner month for gene therapy, as it marked the commercial debut of the first gene therapy to treat an inherited disease – Spark Therapeutics’ Luxturna. The drug treats people living with inherited retinal diseases caused by the RPE65 gene.
This medical milestone was chronicled around the country as patients looked forward to gaining more vision, independence and a new perspective on life. For uplifting reads, check out this coverage of patients named Jack, Creed and Toby.
In other gene therapy news, researchers at Duke Health are seeking funding to launch a clinical trial of an experimental treatment for Pompe disease. The therapy uses a modified virus to deliver a gene that produces GAA, a critical enzyme missing in people with Pompe disease. Without that enzyme, patients can’t metabolize glycogen so it builds up in the muscles, leading to degradation of the tissue. Untreated, the disease can lead to respiratory problems, heart failure and death. The team at Duke has demonstrated the efficacy of their gene therapy in mice but it has not yet been tested in humans.
Voyager Therapeutics, meanwhile, reported positive data from its small Phase 1b trial for a gene therapy to treat Parkinson’s disease, with some participants showing measurable improvement in motor function. Voyager will use the data to set the parameter for dosing in future clinical trials.
Gene therapy wasn’t the only topic of discussion last month. In Europe, researchers used a stem cell patch to restore some vision in two patients with age-related macular degeneration (AMD). The patch, derived from embryonic stem cells, is being evaluated in a Phase 1 clinical study. This good news, while preliminary, is particularly welcome as the industry continues to grapple with unregulated stem cell clinics and treatments that sometimes produce horrifying consequences for patients. A few years ago three patients with macular degeneration were blinded after undergoing an unregulated stem cell procedure meant to improve their vision.
Finally, we turn to CAR-T news. The first two CAR-T therapies — which involve extracting a patient’s immune cells, engineering them to home in on cancer cells, and then returning them to the patient – were approved last year. Patient Nicole Gularte received a CAR-T treatment as doctors said she was near death from leukemia – and made a strong recovery. She shared her story with the Philadelphia Business Journal in a piece that chronicles the highs and lows of treatment over the years and details her journey from hospice to remission.
Investment is still flowing steadily into CAR-T treatments as JW Therapeutics, a company created by WuXi AppTec and Juno Therapeutics (which is now owned by Celgene), raised a $90 million series A funding round. And in early April, a new CAR-T startup, Allogene Therapeutics, burst on the scene with $300 million in investment from a consortium including Pfizer, Vida Ventures and TPG Capital.
Still curious about how CAR-T works as a precision cancer therapeutic? Check out this video from STAT.