We have very exciting news for the LGMD2L community. We've partnered with Cure Rare Disease (CRD), a nonprofit life sciences company, to develop a gene replacement therapy.

CRD was founded to create treatments for rare diseases that receive little attention from traditional pharmaceutical companies. Their pipeline already includes gene replacement therapies for two related conditions, LGMD2I/R9 and LGMD2G/R7 — and as of today, LGMD2L/R12 is officially part of that effort. Because CRD is already working on similar forms of LGMD, they can build on existing research, tools, and manufacturing processes to accelerate development of our therapy. Their deep experience in this area gives us strong confidence that they can move quickly while maintaining the highest standards of safety and rigor.

Our scientific advisors believe that gene replacement therapy represents the best chance of finding a treatment for LGMD2L in the near future. Compared to other therapeutic approaches, gene therapies are well understood and well tested, and regulatory bodies like FDA have approved gene therapies for related diseases. A gene therapy works by introducing the missing gene into the patient's cells, typically using a virus that has been modified to remove its harmful DNA and replace it with the needed gene. All of CRD's therapeutic candidates use a next-generation delivery vector, which has the potential to be safer than the viral vectors used in gene therapies developed in the past.

Since Sarepta announced in July 2025 that it was discontinuing most of its gene therapy pipeline, no companies have been actively developing a therapy for LGMD2L. This partnership marks a pivotal moment for our community. Not only is there now active work on a treament, but it is led by nonprofit, patient-driven organizations whose sole mission is to help patients as quickly as possible.

We've already managed to raise over $150,000 from private donors to cover the cost of this research for at least the next six months. To cover the six months after that, we'll need to raise a further $200,000. At the end of the first year, we hope to already have a candidate therapy and to have finished the preclinical animal research that is needed to apply for approval to run a human clinical trial. As always, we rely on your support -- anything you can donate will have a real impact on our ability to create this therapy. Please spread the word!

Read the full press release here: https://www.businesswire.com/news/home/20260302184269/en/Cure-Rare-Disease-and-LGMD2L-Foundation-Announce-Multi-Year-Partnership-to-Develop-Gene-Therapy-for-Anoctamin-5-Related-Disease