Facioscapulohumeral Muscular Dystrophy 1B

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.

SOLVE FSHD contributes more than $1 million to n-Lorem to support research efforts to inform a therapeutic strategy for FSHD2 nano-rare patients SAN DIEGO--(BUSINESS WIRE)-- n-Lorem, a nonprofit foundation, today announced that Solve FSHD has provided one million dollars in support of n-Lorem’s efforts to fully understand the complex molecular cause of facioscapulohumeral muscular dystrophy-2 (FSHD2). This research will be critical to identifying a strategy for treatment for FSHD2 patients. In addition, this funding supports the expansion of n-Lorem’s research and discovery efforts, significantly enhancing the capabilities of the foundation. “We are grateful to Solve FSHD for this generous support. Not only do we hope to better understand FSHD2, but we hope to identify a path forward for a therapeutic strategy for these patients,” said Stanley T. Crooke, M.D., Ph.D., Founder, CEO and Chairman of n-Lorem Foundation. “Additionally, as we invest in expanding our research capabilities, we enhance the efficiency and capacity of our drug discovery and development programs, which will benefit all of our patients today and in the future.” FSHD2 is a form of muscular dystrophy that affects approximately five percent of FSHD patients. FSHD2 has been linked to mutations on a gene called SMCHD1, but other FSHD2 genes have been identified and approximately two percent of FSHD cases have an unknown genetic cause. The molecular complexity of FSHD2 makes the development of an effective treatment extremely challenging. “We are pleased to be partnering with n-Lorem Foundation to gain further insight into the molecular mechanisms that lead to FSHD2. Until the molecular cause of FSHD2 is better understood, progress on a potential therapeutic is greatly disadvantaged. n-Lorem is the only non-profit with access to three decades of learning in the RNA-targeted therapeutic space and the scientific team to aggressively pursue this complex disease,” said Dr. Eva Chin, Executive Director of Solve FSHD. “Our hope is that this funding will further our understanding of FSHD2, but also other programs, patients and their families,” added Chip Wilson, Founder and Chairman of the Board for Solve FSHD. “We are extremely grateful for the pioneering work of Dr. Crooke and his talented team of scientists at the Foundation.” Learn more about n-Lorem’s mission at , and please consider giving to n-Lorem to bring hope, possibility and treatment options to these needy patients and families. Subscribe: n-Lorem Patient Empowerment Program Podcast Series Watch: n-Lorem Foundation Delivers Hope and Treatment for Patient with Nano-Rare Disease Watch: n-Lorem Foundation: Creating a Better Future for Nano-Rare, One Patient at a Time SOLVE FSHD Solve FSHD is a Vancouver-based venture philanthropic organization established to catalyze innovation and accelerate key research in finding a cure for facioscapulohumeral muscular dystrophy (FSHD). FSHD is one of the most prevalent forms of muscular dystrophy, and the organization will work as a catalyst to accelerate drug developments and treatments for FSHD2 while further benefiting FSHD1. 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. The organization will fund innovative biotech and biopharma research and development activities that accelerate novel treatments and therapies for FSHD. About n-Lorem n-Lorem Foundation is a non-profit organization established to apply the efficiency, versatility and specificity of antisense technology to charitably provide experimental antisense oligonucleotide (ASO) medicines to treat nano-rare patients diagnosed with diseases that are the result of a single genetic defect unique to only one or very few individuals. Nano-rare patients describe a very small group of patients (1-30 worldwide) who, because of their small numbers, have few if any treatment options. n-Lorem Foundation was created to provide hope to these nano-rare patients by developing individualized ASO medicines, which are short strands of modified DNA that can specifically target the transcripts of a defective gene to correct the abnormality. The advantage of experimental ASO medicines is that they can be developed rapidly, inexpensively and are highly specific. To date, n-Lorem has assisted in the development and treatment of 14 nano-rare patients and received over 150 applications for treatment with more than 70 nano-rare patients approved. n-Lorem was founded by Stanley T. Crooke, M.D., Ph.D., former chairman and CEO of Ionis Pharmaceuticals, who founded Ionis Pharmaceuticals in 1989 and, through his vision and leadership, established the company as the leader in RNA-targeted therapeutics. 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