According to a story from BioSpace, the gene therapy companies Sarepta Therapeutics, Inc. and StrideBio, Inc. have recently announced the completion of a licensing and collaboration agreement that could lead to the development of several gene therapies for a variety of rare genetic disorders. As a part of this deal Sarepta will have exclusive license to StrideBio’s capsid technology. Capsids offer an improved method for developing gene therapies that use viral vectors for the delivery of genetic material. Advantages include greater potency, the ability to avoid antibodies from the immune system, and tissue tropism. The partners also aim to collaborate on solutions to re-dosing for patients that are being treated with adeno-associated virus (AAV)-based gene therapies.
Terms of Partnership
Under the agreement, StrideBio’s primary responsibility will be the development of capsids and product candidates, as well the manufacture of gene therapies that are ultimately selected for testing in the clinical setting. Some of this responsibility will also be shared with Sarepta, but this company’s primary role will be commercialization and late-stage development. Potential rare diseases that will be targeted by the partners include Niemann-Pick disease, Rett syndrome, Angelman syndrome, and Dravet syndrome. However, there is potential for up to four additional disorders to be targeted as well. These sorts of partnerships are intended to combined the greatest relative strengths and capabilities of the participant companies. This allows them to achieve milestones that they couldn’t reach on their own or in a much quicker time frame.
About Gene Therapies
Gene therapies have gained a lot of attention in recent years as an innovative approach that has the potential to lead to major breakthroughs in the treatment of a wide variety of genetically-linked diseases and disorders. This has even more significant implications for the field of rare diseases, as many of the rarest syndromes are the result of genetic mutations. Currently, only a small number of gene therapies have achieved market approval, but they have had significant impact, and many new therapies are in development in the experimental stage.
Hopefully this partnership will result in the creation of new and successful gene therapies for a variety of rare genetic disorders.
