AskBio develops and manufactures AAV gene therapies for the treatment of patients with rare genetic diseases. Credit: Mark Warner.

Gene therapy company Asklepios BioPharmaceutical (AskBio) has received an investment of $225m from TPG Capital and Vida Ventures to accelerate its long-term growth.

In addition, the company founders and board members are co-investing an additional $10m. The investment gives TPG Capital and Vida Ventures a minority stake in AskBio.

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Founded in 2011, AskBio develops and manufactures adeno-associated virus (AAV) gene therapies for the treatment of patients suffering with rare genetic diseases.

The company plans to use the funds to drive clinical trials, boost manufacturing capabilities and expand its capacity.

AskBio co-founder and CEO Sheila Mikhail said: “With the funding from TPG and Vida, we will be able to accelerate our development of a broad range of transformative therapies for those affected by serious and oftentimes incurable genetic diseases.

“We look forward to advancing our approaches for repeat administration and avoidance of neutralising antibodies into the clinic to maximise the number of patients who benefit from AAV therapies.”

“We look forward to advancing our approaches for repeat administration and avoidance of neutralising antibodies into the clinic to maximise the number of patients who benefit from AAV therapies.”

The company’s gene therapy platform comprises an AAV cell line manufacturing process called Pro10, which is leveraged by Pfizer, Takeda and Viralgen Vector Core.

AskBio is also engaged in developing gene therapies to treat heart failure, haemophilia, Duchenne Muscular Dystrophy, Limb Girdle 2i, Pompe disease, Myotonic Dystrophy and additional muscular and neurological disorders.

Its portfolio company Actus Therapeutics is working on Pompe disease therapeutics, while another portfolio firm NanoCor Therapeutics collaborated with Medtronic on a gene therapy for congestive heart failure.

Baxter Therapeutics acquired AskBio’s special purpose entity (SPE) Chatham Therapeutics in 2014.  Chatham leveraged AskBio AAV platform to treat haemophilia. These programmes are now owned by Takeda.

In 2016, AskBio sold its Bamboo Therapeutics, which was focused on using the AAV platform for Duchenne Muscular Dystrophy, to Pfizer.

Recently, the gene therapy company collaborated with Touchlight Genetics to commercialise a safe, and cost-effective alternative to plasmid DNA.