Santhera has entered into an agreement with SEAL Therapeutics, a spin-off company from the Biozentrum of the University of Basel, which will further develop a gene therapy approach intended for the treatment of LAMA2-deficient congenital muscular dystrophy. LAMA2 is an inherited and severe form of muscular dystrophy that affects children and for which there is currently no treatment.
SEAL Therapeutics will advance the gene therapy technology building on existing research progress from the Biozentrum Basel and Rutgers, both previously partially funded by Innosuisse and/or Santhera. The new company aims to team up with and support a pharmaceutical partner with experience in gene therapy technologies for clinical development and registration with the ultimate goal to make this innovative treatment approach available to patients with LAMA2 MD and their families.
Previous license agreements and preclinical collaborations that existed between Santhera and University Basel as well as Rutgers, The State University of New Jersey, have been terminated. The new arrangement allows Santhera to focus on its core clinical assets vamorolone and lonodelestat, freeing up resources to advance these platform compounds towards registration and market entry.
Founders of SEAL Therapeutics are Prof. Markus Rüegg and Dr. Judith Reinhard at the Biozentrum and Dr. Thomas Meier, former CEO of Santhera Pharmaceuticals. “The preclinical results in mouse models for LAMA2 MD are very promising,” said Prof. Markus Rüegg, Co-Founder and CEO of SEAL Therapeutics. “Our data clearly demonstrate that simultaneous expression of two specially designed linker proteins leads to sustained improvement in muscle histology, increased muscle mass and strength, improved body weight, and resulted in a remarkable increase in lifespan compared to untreated animals. We now aim to translate this technology for use in human LAMA2 MD patients as soon as possible.”
Supported by the Swiss Foundation for Research on Muscle Diseases
SEAL Therapeutics and the Swiss Foundation for Research on Muscle Diseases have teamed-up in their desire to make this innovative treatment approach available to LAMA2 MD patients as soon as possible. The foundation has invested in SEAL Therapeutics to support the teams' efforts towards clinical development and registration.
LAMA2-related muscular dystrophy (LAMA2 MD or MDC1A) is one of many genetic forms of muscular disease, which typically lead to profound muscle loss, weakness, loss of ambulation and ultimately life-threatening complications. LAMA2 MD is particularly severe, often affecting children as infants. The disease is rare and there is currently no therapy available.
(Press release / SK)
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