Orchard gets $20M to trial ‘bubble boy’ gene therapy

CIRM is putting up $20 million to trial the rare disease gene therapy

Orchard Therapeutics has secured a $20 million grant to trial its stem cell gene therapy in patients with severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID). The funding will enable Orchard to learn whether its lentiviral approach can offer patients an alternative to GlaxoSmithKline's Strimvelis.

The California Institute for Regenerative Medicine (CIRM) is putting up the cash for the clinical trial, which Orchard is working on with the UCLA Broad Stem Cell Research Center. UCLA is one of two sites with experience administering the autologous ex-vivo lentiviral gene therapy. The other is Great Ormond Street Hospital in London, U.K..

These sites have now treated 40 patients, giving Orchard cause for optimism about the safety and efficacy of the approach. People with ADA-SCID have mutations that cause a shortage of white blood cells. This makes the patient vulnerable to infections, resulting in babies dying within months of being born unless treated. The 40 patients treated with the gene therapy are all still alive.


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To create the gene therapy, a copy of a missing or faulty gene is inserted into stem cells taken from a patient. By administering these cells into the patient, Orchard thinks it can restore immune function.

Similar thinking underpins GSK’s Strimvelis. GSK won approval for its ADA-SCID gene therapy Strimvelis in Europe earlier this year. But, while the presence of a competitor in an indication that affects 350 people a year could be seen as reason to back away from the niche, Orchard thinks it can take market share.

The optimism is based on a belief that Orchard’s lentiviral gene therapy is safer than GSK’s gammaretrovirus-based approach. Gamma retroviruses have been linked to safety scares, although Strimvelis has a clean track record.

Orchard has nonetheless persuaded some notable names to bankroll its endeavors. The company emerged in May with $33 million in funding from a Series A round led by F-Prime Capital. Orchard went on to set up a U.S. base in Foster City, California, setting it up to pull in cash from CIRM.

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