New York, Oct. 24, 2024 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) proudly announces that its groundbreaking MDA Kickstart program has received both an FDA Rare Pediatric Disease designation and Orphan Drug Designation for congenital myasthenic syndrome caused by CHAT (choline acetyltransferase) gene deficiency. This major milestone helps to incentivize and support the development of gene therapies targeting ultra-rare neuromuscular diseases, which often lack commercial incentives but represent high unmet medical needs.

About MDA Kickstart
The MDA Kickstart program began in 2023 and launched its first project with UC Davis and biomanufacturing leader Forge Biologics in September 2024. The program is a first-of-its-kind initiative to address the therapeutic gap for neuromuscular diseases affecting fewer than 1,000 people in the U.S. It aims to streamline the path from early research to clinical trials, de-risking and expediting the development of gene therapies for rare conditions. The first project within the MDA Kickstart program is focused on congenital myasthenic syndrome due to mutations in the choline acetyltransferase gene (CHAT) and likely affects less than 250 people in the US.

"Receiving these important designations from the FDA for our pilot MDA Kickstart indication is an essential first step in supporting our ability to bring life-changing treatments to patients living with this rare disease," said Sharon Hesterlee, PhD, Chief Research Officer, Muscular Dystrophy Association. "This is a critical step toward fulfilling our mission to ensure that no patient is left behind in the era of genetic medicines."

Collaboration with UC Davis
The first project under MDA Kickstart focuses on a form of Congenital Myasthenic Syndrome (CMS) caused by mutations in the CHAT gene. This rare genetic condition leads to severe muscle weakness and potentially fatal episodes of respiratory failure. Developed by Ricardo A. Maselli, MD, at UC Davis, the gene therapy aims to replace the defective CHAT gene, offering hope to patients who currently have no approved treatment options.

“For many of the young patients that I see in the clinic battling CMS, these designations are a crucial first step towards developing an effective therapy. These designations unlock a pathway to treatments that can profoundly change their lives and it's nice to see the FDA acknowledge CMS as a condition of unmet need,” said Dr. Maselli.

Manufacturing Partnership with Forge Biologics
MDA has also partnered with Forge Biologics to provide AAV gene therapy process and analytical development manufacturing services for the Kickstart program. The partnership will leverage Forge’s proprietary technology, and all development and manufacturing activities will occur in Forge’s 200,000-square-foot cGMP facility in Columbus, Ohio.

"We are excited to support the MDA Kickstart Program and help advance these critical projects to the next stage of development through our leading AAV manufacturing expertise and services," said John Maslowski, President and Chief Executive Officer at Forge. Collaborating with MDA embodies our mission at Forge to help deliver potentially life-changing treatments to patients."

FDA Priority Review Voucher Reauthorization
The Rare Pediatric Disease Designation granted to the MDA Kickstart program for CMS is part of a federal incentive program designed to encourage the development of therapies for rare diseases affecting children. However, the Priority Review Voucher program is set to expire on December 20, 2024, unless Congress moves to reauthorize it.

"We’re proud to have made an impact with advocates for the reauthorization of the Rare Pediatric Disease PRV program as it is essential for continuing progress in rare disease treatment," said Paul Melmeyer, Executive Vice President of Public Policy and Advocacy, Muscular Dystrophy Association. "On September 10, an MDA contingent of more than 100 advocates urged lawmakers to extend this program so that more therapies, like the ones being developed through MDA Kickstart, can reach the patients who desperately need them. Their voices were heard and we’re extremely proud of this progress.”

“These designations enable us to synchronize a development plan with regulatory initiatives and incentives at an early stage.” says Marina Kolocha, MDA Kickstart Project Manager.

About Muscular Dystrophy Association
Muscular Dystrophy Association (MDA) is the #1 voluntary health organization in the United States for people living with muscular dystrophy, ALS, and over 300 related neuromuscular conditions. For nearly 75 years, MDA has led the way in accelerating research, advancing care, and advocating for the support of our families. MDA's mission is to empower the people we serve to live longer, more independent lives. To learn more visit mda.org and follow MDA on Instagram, Facebook, X, Threads, TikTok, LinkedIn, and YouTube.

About UC Davis Institute for Regenerative Cures
UC Davis is playing a leading role in regenerative medicine, with nearly 150 scientists working on a variety of stem cell-related research projects at campus locations in both Davis and Sacramento. The UC Davis Institute for Regenerative Cures, a facility supported by the California Institute for Regenerative Medicine (CIRM), opened in 2010 on the Sacramento campus. This $62 million facility is the university's hub for stem cell science. It includes Northern California's largest academic Good Manufacturing Practice laboratory, with state-of-the-art equipment and manufacturing rooms for cellular and gene therapies. UC Davis also has a Translational Human Embryonic Stem Cell Shared Research Facility in Davis and a collaborative partnership with the Institute for Pediatric Regenerative Medicine at Shriners Hospital for Children Northern California. All of the programs and facilities complement the university's Clinical and Translational Science Center and focus on turning stem cells into cures. For more information, click here.

About Forge Biologics
Forge Biologics, a member of Ajinomoto Bio-Pharma Services, is a hybrid gene therapy contract manufacturing and clinical-stage therapeutics development company, enabling access to life-changing gene therapies by bringing them from concept to reality. Forge’s 200,000 square foot facility, the Hearth, is headquartered in Columbus, Ohio, and houses 20 custom-designed cGMP suites with 20,000L of bioreactor capacity. Forge’s end-to-end, scalable plasmid and AAV manufacturing services include research-grade manufacturing, process and analytical development, cGMP manufacturing, fill and finish, and integrated regulatory support to help accelerate the timelines of transformative medicines for patients with genetic diseases. To learn more, visit www.forgebiologics.com.

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• MDA Kickstart Program

Mary Fiance, Vice President, Strategic Communications
Muscular Dystrophy Association
press@mdausa.org