Hope for the Future of Muscular Dystrophy Treatment: Kate Therapeutics Harnesses their myoAAV Technology for FSHD

By Justin Cohen, PhD

Kate Therapeutics, a San Diego base biotechnology company has announced funding to advance
their muscle and cardiovascular disease portfolio. Among the first diseases to be explored is
Facioscapulohumeral muscular dystrophy (FSHD), exciting news for patients like myself and their
families who are impacted from this affliction. Included among these ranks is co-founder and Chief
Scientific Officer, Sharif Tabebordbar PhD, whose father is an FSHD patient and inspiration for this work.

Kate Therapeutics’ platform focuses on improving delivery of gene therapies. Current methods use a
tool called adeno-associated virus (AAV) which basically functions as a truck that delivers its cargo into
cells. They are a popular tool for this purpose as it takes advantage of the fact that viruses ‘want’ to
infect cells and deliver their genetic material. However, there are several challenges with this method
for gene therapy in muscle diseases. First is the amount of AAV that needs to be produced. Current
methods to produce AAV are difficult and costly to scale up to the concentration needed for
administration to a person. This is compounded by the fact that skeletal muscle as a whole is the largest
organ and requires even more of the therapy. Furthermore, larger amounts of AAV increases the risk of
adverse immune reactions that can harm or even kill the patient.

Kate Therapeutic’s platform begins to overcome some of these challenges based on work performed
by Tabebordbar and colleagues at the Broad Institute of MIT. By randomly mutating the portion of AAV
that allows it to target cells, a version was created called MyoAAV that has a much greater affinity for
muscle tissue. This increased specificity for muscle reduces the amount of MyoAAV needed to be
administered, helping with some of the challenges for gene therapy including reducing costs and the
risks of a negative immune response.

While the application of MyoAAV towards muscle diseases are still in the early stages, there is great
potential for its use in FSHD. I and many other patients will be keeping a close eye on Kate Therapeutics
in the future and are greatly excited to see what comes next.

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