Asklepios BioPharmaceutical Secures $235 Million in Equity Financing to Advance AAV Gene Therapies for Rare Diseases

VCBeat (WeChat Official Account: vcbeat) learned from foreign media that on April 11, U.S. local time, Asklepios BioPharmaceutical (hereinafter referred to as “AskBio”), a biotechnology company headquartered in Chapel Hill, North Carolina, secured a total of $235 million in funding. Of this amount, $225 million was provided by investors TPG Capital and Vida Ventures in exchange for partial equity stakes in AskBio, while the remaining $10 million was jointly contributed by AskBio’s founders and board members.

AskBio is a leading AAV gene therapy company, co-founded in 2001 by Dr. Richard Jude Samulski and Dr. Xiao Xiao of Shanghai Medical University. Dr. Samulski was the first scientist to clone AAV, while Dr. Xiao was the first to develop the minidystrophin gene, which facilitates gene therapy for Duchenne muscular dystrophy (DMD).

AskBio’s name is inspired by Asclepius, a figure from Greek mythology who cured a series of incurable diseases and has become a symbol of modern medicine. Like Asclepius, AskBio focuses on rare genetic disorders that lack treatment options, such as Duchenne muscular dystrophy (DMD), Pompe disease, limb-girdle muscular dystrophy, and giant axonal neuropathy (GAN).

Genes serve as the blueprint of the human body, providing instructions to cellular machinery on how to synthesize corresponding proteins. The expression of defective proteins is typically caused by damage to or deletion of the corresponding genes, but may also result from erroneous modifications of downstream gene products. For genetic disorders arising from such protein misexpression, conventional pharmacological treatments usually work by altering the microenvironment surrounding diseased cells to induce a relevant cellular response. In contrast, gene therapy delivers therapeutic genes into diseased cells via viral vectors, thereby directly correcting the underlying genetic defect.

AskBio’s core technology, AAV gene therapy, is a gene therapy approach that uses adeno-associated virus as a vector. AAV stands for recombinant adeno-associated virus, which is non-pathogenic, enables long-term and effective gene expression, and is amenable to genetic engineering. Recombinant adeno-associated virus (AAV) can serve as a primary gene delivery vector, delivering therapeutic genetic material to target cells and curing diseases by providing new replacement genes that encode human therapeutic proteins.

Currently, the company possesses its proprietary Pro10 cell line manufacturing process and a rich AAV capsid library, and has generated hundreds of proprietary third-generation gene vectors. These vectors have entered clinical testing stages, with a series of clinical programs underway for multiple indications, including Pompe disease. Furthermore, the company’s Pro10 cell line manufacturing process has produced 10^18 AAV vector genomes in 200-liter bioreactors, establishing it as the industry-recognized gold standard for scalable manufacturing.

AskBio has partnered with multiple biotechnology companies to jointly develop new therapies for rare genetic diseases. Among these collaborations, it is working with Bamboo Therapeutics to develop therapies for Duchenne muscular dystrophy and other rare diseases; with Chatham Therapeutics to develop novel treatments for hemophilia; and most recently, with Touchlight Genetics to develop alternatives to plasmid DNA.

TPG Capital is a global private equity firm founded in 1992 and headquartered in Austin, Texas, USA. The firm manages $45 billion in assets, with primary investments in the consumer, retail, media, industrial, financial services, natural resources, technology, real estate, and healthcare sectors.

Vida Ventures is a life sciences investment fund established in 2017 and based in Boston, Massachusetts, USA. Co-founded by a group of scientists, physicians, and entrepreneurs, the firm primarily invests in companies focused on medical therapeutics. To date, it has completed 13 investments.

(Compiled by Wang Chan)