Gensaic Receives Funding From the CF Foundation to Develop a Gene Therapy for People With Cystic Fibrosis

2023-01-09
基因疗法
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Gensaic, a company that seeks to reimagine gene therapy, announced today that it has received an investment from the CF Foundation to develop a gene therapy for Cystic Fibrosis (CF).
“The investment by the CF Foundation is a recognition of the alignment between Gensaic’s and the CF Foundation’s mission. Together, we hope to bring forward a gene therapy to treat the underlying cause of this devastating disease for all patients with CF,“ said Lavi Erisson, MD, MPH, MBA, co-founder, and CEO of Gensaic. “Working with the CF foundation represents an incredible opportunity for Gensaic to lean into the CF Foundation’s leadership in fundamental research, patient advocacy, and therapeutic development”.
Under the terms of the 1st tranche, Gensaic will harness its proprietary PDP platform to achieve pre-clinical milestones upon which the 2nd tranche will be transacted. Gensaic will retain all rights to the continued development of a lead program.
About Gensaic
Gensaic, Inc. is a redosable genomic medicines company spun out of MIT and funded in 2021. It is based in Cambridge, MA. Leveraging Nobel Prize-winning research on phage-display and directed evolution, Gensaic is pioneering a novel class of gene-delivery vehicles called Genetically encoded Nanoparticles (GeNPs). Gensaic’s first GeNP vehicle is premised on the evolution of M13 phage-derived particles (PDPs), a highly modular system consisting of 3 engineerable capsids and a designer minimal phage DNA (mpDNA) cargo. M13 originates from the microbiome and had been safely administered to human patients for over a decade. PDPs use phage display for targeted tissue delivery and can package > 20kb of genetic information, nearly any human gene. PDPs benefit from scalable and efficient manufacturing in bacterial culture, holding promise for a new era of accessible and affordable genomic medicines. Gensaic’s current strategic areas of focus for clinical translation are muscle, respiratory, and CNS.
Since its inception, Gensaic has established a deep discovery platform, secured a broad intellectual property portfolio, received recognition through four golden ticket competitions from BioMarin, Astellas, Merck, and Novo-Nordisk; and secured its first R&D collaboration in CNS with Ovid Therapeutics. For more information on Gensaic and PDPs visit: www.gensaic.com
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