Research & Development Santhera advances gene therapy for LAMA2 MD through deal...

Santhera advances gene therapy for LAMA2 MD through deal with Seal Therapeutics


Santhera Pharmaceuticals and Seal Therapeutics’ partnership aims to develop gene therapy for the disease known as LAMA2-deficient congenital muscular dystrophy, or LAMA2 MD. The two pharmaceutical companies have entered into an agreement for this purpose.

Seal Therapeutics has evolved from the University of Basel’s Biozentrum and is utilizing previous research conducted for gene therapy technology. The research had been funded by both Santhera and Innouisse at the research locations of the University of Basel (Switzerland) and Rutgers University (New Jersey).

LAMA2-deficient congenital muscular dystrophy is a result of a gene mutation that affects muscle stability as this mutation of the LAMA2 gene controls the part of the three-dimensional extracellular matrix (ECM)  of the muscles that encompasses it, providing the muscle stabilization. 

With LAMA2-MD, due to gene mutation, the ECM does not develop correctly. As a result, in children with the disease, it is observed that they grow up to have severely weak muscles, muscle atrophy, low muscle tone, and increased difficulty in breathing.

The extracellular matrix (ECM) itself is made of proteins as well as other molecules and problems with it cause muscle fragility.  

Research by Bioznetrum and Rutgers, conducted by Markus Rüegg and Peter Yurchenco at the respective facilities found the potential of linker proteins, which are small protein particles, obtained from the ECM’s proteins in therapeutic treatment of LAMA2-MD.

The protein fragments are used as a replacement for the affected protein due to the gene mutation, and ensure the ECM can function, and thus the muscle can as well. 

Santhera’s previous license agreements with the universities have since been terminated as this partnership with Seal Therapeutics will allow for payments to be made to Santhera, dependent on the future proceeds of Seal Therapeutics. This will allow resources to be funneled towards the registration of the lead products as well as their market entry. 

Santhera and Seal representatives noted that this partnership has allowed both companies to bring together their areas of expertise and develop gene therapy solutions for LAMA2-MD. The partners will build onto the existing research available to SEAL and move towards clinical trials and finally therapy approval in the future.

The linker proteins used to counter the effects of LAMA2-MD utilize the delivery mechanism of an altered adeno-associated virus. The virus is harmless and through its use gene therapy can be administered.

In the animal clinical experiment, this approach remained effective and showed positive results. The linker proteins administered through the virus were able to support the healthy development of the ECM which increased muscle mass and tone in mice, as well as their life span by over five times when compared to mice who had not been given this treatment. 

The SEAL co-founder noted that this experiment on mice showed great promise for replication of results in humans. The preclinical results show that the gene therapy technology for LAMA2-MD has the potential to help treat human patients later on, which the company aims to achieve as quickly as possible.

Seal Therapeutics receives funding from the Swiss Foundation for Research on Muscle Diseases for its work in gene therapy for LAMA2-MD.

A company representative assured that the company will not stray its focus from its key projects like Lonodelestat and Vamorolene, due to the gene therapy partnership with Seal. 

Another drug developed by the company for muscular dystrophy is in a partnership with ReveraGen BioPharma, named Vamorolone. The drug has shown promise in clinical trials and the partners have plans to pursue U.S. FDA therapy approval this month.

Lonodelestat, on the other hand, is being developed as a treatment for a genetic disease known as cystic fibrosis.

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