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Home » gene therapies

Articles Tagged with ''gene therapies''

CAR T cell attacking cancer cells

In CAR T therapy, China excels in speed but quality remains a concern

June 1, 2021
By Elise Mak
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.
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Optogenetics illustration

Beyond Luxturna companies vie to become the next ocular gene therapy

Feb. 22, 2021
By Brian Orelli
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.
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DNA, dollars illustration

Gene therapy surges as risks reduced

Dec. 21, 2020
By Brian Orelli
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.
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Brain and DNA

Eli Lilly acquiring Prevail for up to $1.04B

Dec. 15, 2020
By Brian Orelli
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.
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Skeletal disorder firm Innoskel launches with $24M

Dec. 14, 2020
By Nuala Moran
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.
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Syncona’s kidney disease gene therapy play: Purespring seeks answers to $60M questions

Nov. 19, 2020
By Cormac Sheridan
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.
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Janssen, AGTC report initial 12-month data for gene therapies in X-linked RP

Nov. 13, 2020
By Cormac Sheridan
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.
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FDA vector objector, Bluebird SCD filing pushed out a year

Nov. 5, 2020
By Randy Osborne
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.
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Adenovirus cells

"Foundational" research shows early gene therapy prevents Angelman syndrome

Oct. 30, 2020
By W. Todd Penberthy
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.
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Rays of light beaming from eye

Sparingvision adds $52.5M for gene therapy trial, expansion to U.S.

Oct. 20, 2020
By Nuala Moran
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.
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