RARE Daily

Affinia Therapeutics Raises $60 Million to Advance Gene Therapies Targeting Muscle and CNS Diseases

March 31, 2020

Rare Daily Staff

Affinia Therapeutics has raised $60 million in a series A financing to advance its platform for rationally designed adeno-associated virus vectors and to develop gene therapies for people affected by muscle and central nervous system diseases with significant unmet need.

Seed venture investors F-Prime Capital and New Enterprise Associates co-led the round alongside new investor Atlas Venture, with participation from seed investors Alexandria Venture Investments, Lonza, and Partners Innovation Fund.

Affinia’s executive team includes recently appointed CEO Rick Modi, the former chief business officer of the gene therapy biotech AveXis. Sean Nolan, former CEO of AveXis will serve as chairman of the board of directors. Nolan and Modi were key players in advancing the gene therapy for spinal muscular dystrophy, Zolgensma, to market.

“At Affinia Therapeutics, we’re setting a new standard in gene therapies by leveraging our proprietary platform to methodically engineer novel AAV vectors, and gene therapies that have remarkable targeting properties,” said Modi.

The company’s technology is licensed from Lonza and Massachusetts Eye and Ear, and was developed at the Grousbeck Gene Therapy Center by Luk Vandenberghe, associate professor at Massachussetts Eye and Ear and Harvard Medical School and a co-inventor of AAV9, through a sponsored research agreement with Lonza.

“Affinia Therapeutics is bringing together complementary expertise allowing us to realize a rational design future for AAV vectors, promoters and other components of gene therapies,” said Vandenberghe. “By leveraging synthetic and systems biology combined with high-throughput screening and tissue and single-cell resolution, we are aspiring to achieve much-needed improved pharmacological control of this novel modality in medicine.”

Vandenberghe and his team have developed AAVSmartLibraries, which comprises thousands of functional novel AAV vectors. Each vector is uniquely identified, and the libraries can be screened across species for parameters of high interest, including tissue tropism, manufacturing yield and pre-existing immunity. Observations arising from each library screen provide insights into the vector’s structure-function, enabling the rational design of novel vectors and gene therapies with remarkably improved properties. According to the company, Affinia has potentially the world’s largest library of patented functional AAV vectors.

“Affinia Therapeutics’ methodical process for designing and evaluating vectors is a differentiated approach to gene therapy, and the highly experienced leadership team will help carry these discoveries to the development, manufacturing and commercialization of transformative medicines,” said Ed Mathers, general partner at New Enterprise Associates.

Photo: Affinia CEO Rick Modi

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