Vita Therapeutics Raises $31 Million to Develop Cell Therapies for Neuromuscular Diseases and Cancers

Vita Therapeutics, a cell engineering company developing cellular therapies to treat muscular dystrophies and cancers, said it closed a $31 million series B financing.

Cambrian BioPharma and new investor Solve FSHD led the financing. New investors Riptide Ventures and Cedars Sinai participated in the financing, alongside TEDCO and other existing investors. Proceeds from the financing will be used to advance Vita’s lead pre-clinical program VTA-100 for limb-girdle muscular dystrophy (LGMD2A) to the clinic. It will also fund the development of Vita’s newest program, VTA-120 for the treatment of patients with facioscapulohumeral muscular dystrophy (FSHD), and to further expand Vita’s discovery pipeline. Since inception, Vita has raised a total of $66 million.

“The support from this strategic group of quality investors further validates Vita’s cell therapy platform and our mission to bring transformative therapies that target the root cause of disease to patients with muscle disorders and cancers,” said Douglas Falk, CEO at Vita Therapeutics. “We are making notable progress with our investigational IND-enabling studies for VTA-100 and are on track to reach the clinic with this important therapeutic candidate within 18 months. Additionally, we are excited to further expand our pipeline to include VTA-120 for the treatment of patients with FSHD.”

‍Limb-girdle muscular dystrophy (LGMD) is a group of disorders that cause weakness and wasting of muscles closest to the body (proximal muscles), specifically the muscles of the shoulders, upper arms, pelvic area, and thighs. The severity, age of onset, and disease progression of LGMD vary among the more than 30 known sub-types of this condition and may be inconsistent even within the same sub-type. As the atrophy and muscle weakness progresses, individuals with LGMD begin to have trouble lifting objects, walking, and climbing stairs, often requiring the use of assistive mobility devices. There is currently no cure for LGMD, with treatments limited to supportive therapies such as corticosteroids.

‍Facioscapulohumeral muscular dystrophy (FSHD) is an autosomal dominant muscular dystrophy, although thirty percent of new FSHD patients have no prior family history of the disease and result from a congenital spontaneous genetic mutation. FSHD typically first presents with weakness of the muscles of the facial muscles and scapular region, with proximal weakness of the pectoral and abductor muscles limiting upper extremity function at the shoulder girdle. Onset is typically in the teenage and early adult years, but it can present in infancy, which tends to be a more aggressive course. The disease is slowly progressive and approximately 20 percent of patients are wheelchair bound by age 50. Currently there are no treatments specifically indicated for use in FSHD, with no disease-modifying treatments available.

Chip Wilson, founder of lululemon athletica, founded the venture philanthropy Solve FSHD in March, kickstarting it with $100 million to find a cure for FSHD. “Living with FSHD for over 30 years, my upper body muscles are quite wasted. We are hopeful that Vita’s cell therapy approach will stimulate muscle regeneration and help people like me to build up muscle faster than it breaks down,” he said.

‍Vita Therapeutics’ current pipeline includes lead program, VTA-100 for the treatment of LGMD2A, VTA-120 for the treatment of FSHD, and VTA-300 targeting multiple cancers. VTA-100 is currently undergoing investigational IND-enabling studies and is designed to be an autologous treatment that combines gene correction and induced iPSC technology to help repair and replace muscle cells for people with LGMD2A. VTA-120 is designed to be an autologous treatment that combines gene correction and iPSC technology to help repair and replace muscle cells for the treatment of FSHD. VTA-300 is an immunotherapy currently under development that combines proprietary chimeric antigen receptor (CAR) technology and gene editing to target an undisclosed indication.

Douglas Falk, CEO at Vita Therapeutics

Author: Rare Daily Staff