China is making strides in cell and gene therapy, notably so with a 61% surge in the number of clinical trials in six years, a new report by Ernst & Young showed. While analysts noted the increasing innovation efforts and cross-border collaborations, concerns remain if quality will be compromised by speed.
The 2017 FDA approval of Luxturna (voretigene neparvovec-rzyl, Roche Holding AG) spurred a race to create the next gene therapy for the eye. The organ is very amenable to gene therapy given that it's a confined space with post-mitotic cells that has immune privilege and requires substantially smaller amounts of viral vector compared to systemic treatments.
Money is flowing into gene therapy as investors have started looking favorably on the risk-reward prospects of the space. "I believe the risk has really substantially decreased as regulatory path has become clearer. There are more projects with lower risk," said Sarah Bhagat, a partner at Sofinnova Investments.
Prevail Therapeutics Inc. won't be prevailing on its own. Eli Lilly and Co. is acquiring the gene therapy company for $880 million up front plus an earn-out of up to $160 million if Lilly can gain regulatory approval for at least one of Prevail's drugs.
LONDON – Innoskel has arrived on the scene with a €20 million (US$24.3 million) series A funding to advance development of gene therapies for type 2 collagenopathies, a group of rare skeletal disorders that affect the structure of connective tissues, leading to short stature.
DUBLIN – Health care investor Syncona Ltd. has founded a new startup, Purespring Therapeutics Ltd., to take gene therapy into the kidney. It is committing £45 million (US$59.6 million) in series A funding, which will support the build-out of the new company and take at least one program into the clinic.
DUBLIN – The contest to bring a safe and effective gene therapy for X-linked retinitis pigmentosa (XLRP) to market is intensifying. Two of the three contenders with clinical-stage programs reported initial 12-month data from phase I/II trials and are now looking ahead to pivotal trials and beyond.
Shares of Bluebird Bio Inc. (NASDAQ:BLUE) sank 16.6%, or $9.72, to close at $48.83 as Wall Street reacted to news that the U.S. regulatory filing for Lentiglobin in sickle cell disease (SCD) will be delayed. Previously expected in the second half of next year, the filing won’t happen until late 2022.
Scientists working at the University of North Carolina, Chapel Hill reported in the Oct. 21, 2020, issue of Nature on the successful development of a one-time specific sequence-directed gene therapy approach using the combination of AAV with CRISPR technology that successfully prevented the presentation of Angelman syndrome throughout the lifetime of a mouse model.
LONDON – Sparingvision SAS has raised €44.5 million (US$52.5 million) to advance SPVNo6, a gene therapy designed to be effective for all retinitis pigmentosa patients regardless of the underlying mutation, with a phase I dose-ranging study now due to start in 2021.
