VTA-100

Earlier this year, Lululemon’s founder Chip Wilson went public with his rare disease diagnosis of facioscapulohumeral muscular dystrophy type 2, or FSHD type 2, in which the muscles degenerate over time. Wilson also committed $100 million to find a cure for FSHD through a new venture, Solve FSHD. Chip Wilson Now, up to $10 million of that commitment is going to Vita Therapeutics, a Baltimore-based cell therapy biotech, and Vita will be adding a discovery-phase FSHD cell therapy to its pipeline. Solve FSHD’s executive director Eva Chin will also be joining Vita’s board. The money from Solve FSHD is part of Vita’s $31 million Series B. Cambrian BioPharma, which launched Vita, also led this round of funding, followed by Riptide Ventures, Cedars Sinai, TEDCO, and others. Last year, Cambrian, the anti-aging portfolio company headed by atai co-founder Christian Angermayer, unveiled Vita with a $32 million Series A. UPDATED: Can a cell therapy treat muscular dystrophy? A German billionaire's anti-aging startup is trying to find out Most of the new cash infusion will go toward Vita’s lead program, dubbed VTA-100. The candidate is a cell therapy for limb-girdle muscular dystrophy type 2a (LGMD2A), the most common type of LGMD. While it’s still in preclinical studies, Vita’s CEO Doug Falk said the company hopes to reach the clinic within 18 months. “We’re really on the verge of kicking off the IND-enabling studies. In parallel to those studies, we’re going to be doing the GMP manufacturing that’s needed to go ahead and file an IND,” Falk told Endpoints News . And aside from its muscular dystrophy programs, Vita has an immunotherapy candidate, VTA-300, that it hopes to bring up from discovery to the preclinical stage as well, Falk noted. And it will be looking to add 10-15 employees to its current headcount of around 30. Sarepta is developing a number of gene therapies for LGMD, though its candidate for type 2a specifically is likewise in preclinical studies. But its LGMD candidate SRP-9003 in clinical studies has shown promising early results so far. Sarepta reveals positive results in limb-girdle gene therapy trial. What does that mean for DMD? On the FSHD front, previous attempts to treat the disease have seen little success. In 2019, Acceleron scrapped its program after patients didn’t have functional improvements on the drug. And Fulcrum’s attempt with a drug targeting DUX4, licensed from GSK, failed a Phase II study — though the biotech is pushing onward with Phase III. But aside from Vita, there are a handful of early candidates. Just last week, Sanofi licensed an antibody-RNA conjugate for FSHD from miRecule, an NIH vet-led startup which also got $1 million from Solve FSHD. And Dyne Therapeutics has its own DUX4-targeted preclinical candidate.

- Fundraising led by Cambrian BioPharma and Solve FSHD - - Company expands pipeline with new program, VTA-120, for the treatment of facioscapulohumeral muscular dystrophy (FSHD) - BALTIMORE--(BUSINESS WIRE)-- Vita Therapeutics, a cell engineering company harnessing the power of genetics to develop novel cellular therapies to treat muscular dystrophies and cancers, today announced the completion of a $31 million Series B financing. The fundraise was led by Cambrian BioPharma and new investor Solve FSHD. New investors included Riptide Ventures and Cedars Sinai, which participated 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, MS, Chief Executive Officer at Vita Therapeutics. “This syndicate’s confidence in our ability to further progress our programs is energizing and we are thrilled to have them as partners. 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. I’m incredibly proud of our entire team and the steady momentum we continue to have.” Chip Wilson, Founder of lululemon athletica and of Solve FSHD noted, “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.” “Currently there are no treatments available for FSHD, and there is an urgent need to develop disease-modifying treatments that not only regenerate muscle but correct the genetic defect that otherwise leads to the muscle’s inability to repair itself,” added Eva Chin, Executive Director for Solve FSHD. “We are pleased to support Vita as they continue to expand their induced pluripotent stem cell (iPSC) technology towards FSHD and LGMD.” “Vita Therapeutics aligns with Cambrian’s mission of building medicines that will redefine healthcare in the 21st century,” said Cambrian BioPharma Chief Executive Officer, James Peyer, PhD. “The team, as well as the scientific platform, continues to impress us as they aim to solve for treatments that go beyond symptom management to truly impact these diseases in a positive way.” Pipeline Overview 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. About Limb-Girdle Muscular Dystrophy 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. About Facioscapulohumeral Muscular Dystrophy 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% 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. About Vita Therapeutics Vita Therapeutics is a biotechnology company developing state-of-the-art cellular therapeutics for the treatment of debilitating neuromuscular diseases and cancers. Vita Therapeutics uses induced pluripotent stem cell (iPSC) technology to engineer specific cell types designed to replace those that are defective in patients. The Company is progressing its lead program VTA-100 for the treatment of limb-girdle muscular dystrophy (LGMD2A) with the goal of filing Investigational New Drug Applications with the US Food and Drug Administration in the next 18 months. Long term, the Company is developing its pipeline of cellular therapies following a dual development strategy beginning with autologous-derived cells before moving to a universal hypoimmunogenic cell line. Vita Therapeutics is currently working with numerous partners, including PanCella, Wyss Institute, and Johns Hopkins University, to advance their clinical programs. Learn more about the company at . About Cambrian BioPharma Cambrian BioPharma is building the medicines that will redefine healthcare in the 21st century – therapeutics to lengthen healthspan, the period of life spent in good health. As a Distributed Development Company, Cambrian is advancing multiple scientific breakthroughs each targeting a biological driver of aging. Its approach is to develop interventions that treat specific diseases first, then deploy them as preventative medicines to improve overall quality of life during aging. For more information, please visit or follow us on Twitter @CambrianBio and LinkedIn. About SOLVE FSHD SOLVE FSHD is a venture philanthropic organization established to catalyze innovation and accelerate key research in finding a cure for FSHD. Established by renowned Canadian entrepreneur and philanthropist Chip Wilson, the founder of technical apparel company lululemon athletica inc. Chip has committed $100 million to kick-start funding into projects that support the organization’s mission to find a cure for FSHD by 2027. The goal of SOLVE FSHD is to find a solution that can stop muscle degeneration, increase muscle regeneration and strength, and improve the quality of life for those living with FSHD. For more information, please visit: .