Muscular Dystrophy, Emery-Dreifuss

THURSDAY, Sept. 12, 2024 -- The overall second primary malignancy (SPM) point estimate was 5.8 percent for patients receiving chimeric antigen receptor (CAR) T-cell therapy, according to a study published online Sept. 11 in Clinical Cancer Research . Tobias Tix, from LMU Klinikum in Munich, and colleagues conducted a systematic review to delineate the frequency and subtypes of SPMs following CAR T-cell therapy in lymphoma and myeloma. A total of 326 SPMs were identified across 5,517 patients from 18 clinical trials and seven real-world studies. The researchers found that the overall SPM point estimate was 5.8 percent with a median follow-up of 21.7 months. There were associations for SPM estimates with treatment setting (clinical trials > real-world settings), follow-up duration, and number of previous treatment lines. In a meta-regression model, these associations were confirmed as independent study-level risk factors for SPM. The risk for SPM was similar with either treatment strategy in a subgroup meta-analysis of four trials that randomly assigned patients to CAR T-cell therapy versus standard of care. In an analysis of SPM subtypes, the most common were hematologic malignancies, followed by solid tumors and nonmelanoma skin cancers (37, 27, and 16 percent, respectively). A small minority of events were T-cell malignancies (1.5 percent). "By understanding the factors contributing to SPM development and implementing effective monitoring strategies, health care providers can optimize patient outcomes and ensure the long-term safety of CAR T-cell therapies," the authors write. Several authors disclosed ties to the pharmaceutical industry.

VANCOUVER, British Columbia, April 18, 2023 (GLOBE NEWSWIRE) -- SOLVE FSHD, a venture-philanthropic organization catalyzing the pace of innovation to accelerate a cure for FSHD, announced today an investment in Epic Bio, a biotechnology company focused on the epigenome of complex diseases. Epic Bio is developing novel therapies that dynamically control gene expression to treat these diseases using compact, non-cutting dCas proteins. This investment will support development of Epic’s lead program EPI-321, a novel therapeutic that is designed to correct the epigenetic alterations in the D4Z4 region of chromosome 4. Epic’s unique approach to correct the molecular pathology implicated in Facioscapulohumeral Muscular Dystrophy (FSHD) should lead to an effective treatment that prevents further muscle degeneration and thereby slows disease progression. “SOLVE FSHD is very pleased to partner with Epic Bio and to add them to our diverse portfolio of companies rapidly moving forward potential therapies for FSHD,” stated Eva Chin, Executive Director of SOLVE FSHD. “SOLVE FSHD identified Epic Bio as a company committed to finding a cure for this debilitating condition and we were impressed by the unique approach that the company is taking to alter the epigenetic cause of the disease. We expect that the support from SOLVE FSHD will allow Epic Bio to rapidly progress EPI-321 into clinical trials.” “Epic Bio welcomes the investment by SOLVE FSHD and we appreciate their important support for the future development of EPI-321,” added Amber Salzman, Chief Executive Officer of Epic Bio. “SOLVE FSHD is rapidly becoming the partner of choice for companies developing FSHD treatments, so their investment provides strong validation of the Epic Bio GEMS platform and the EPI-321 program.” “Today we are very happy to add another company to the SOLVE FSHD portfolio,” noted Chip Wilson, founder and chairman of the Board of SOLVE FSHD. “More importantly, EPI-321 represents another potential treatment for FSHD, a devastating condition that I and many others like me suffer with every day.” Terms of the investment were not disclosed. 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 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, widely known as the founder of lululemon and part owner of Amer Sports which holds renowned brands such as Arc’teryx, Salomon and Wilson Sports. 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. If you have FSHD and want to find out about clinical trials or be included in the FSHD registry, please see Solve FSHD’s website - About Epic Bio Epic Bio is a leading epigenetic engineering company, leveraging the power of CRISPR without cutting DNA. The company is using its proprietary Gene Expression Modulation System (GEMS) to develop therapies. Through the company’s library of the most compact Cas DNA-binding proteins to work on human cells, the company is developing in vivo therapies with delivery via a single AAV vector. Epic Bio has an initial focus on Facioscapulohumeral Muscular Dystrophy (FSHD) and is conducting additional research to address Antitrypsin Deficiency (A1AD), Heterozygous Familial Hypocholesterolemia (HeFH), as well as other indications. The company is financially backed by Horizons Ventures and other leading investors. Visit for more information or follow us on Twitter and LinkedIn. Media Contact Swati Mehta PR Associates smehta@prassociates.com 403-804-0768

- 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: .