At the end of November, the Kurt+Peter Foundation met with both Myonexus and Sarepta. Myonexus is projecting a 2020 start for a Phase 1/2 clinical trial for LGMD2C, with the qualification that the schedule remains fluid because of manufacturing capacity. We will continue to update the LGMD2C community as we learn more about Myonexus’s plans for the clinical trial.
We have discussed proposals from Dr. McNally at Northwestern University for the Kurt+Peter Foundation to fund(i) further work on the LGMD2C exon skipping program and (ii) a study of the impact of an alternative steroid dose regimen on LGMD2C patients. Kurt and Peter have started as the first two patients in the alternative steroid regimen study.
The Kurt+Peter Foundation discussed with Northwestern University and the University of Chicago avenues to further the LGMD2C exon skipping program.
The Kurt+Peter Foundation participated in a Myonexus Therapeutics Patient Advisory Board telephone conference with senior management of Myonexus Therapeutics and learned about Myonexus’s plans for engagement with the LGMD community.
Exciting news! Sarepta Therapeutics, Inc. announced that it has entered into a partnership with Myonexus Therapeutics that will allow Myonexus Therapeutics to fund further development, including manufacturing for various forms of Limb-girdle muscular dystrophies (LGMDs), including LGMD2C. As described here, Sarepta will make an upfront payment of $60 million and additional development-related milestone payments.
More exciting news! Elizabeth McNally and Eugene Wyatt at Northwestern University published this paper describing how exon skipping works in LGMD2C. The publication of this peer-reviewed article helps further the exon skipping program. The research was exclusively funded by the Kurt+Peter Foundation.
The Kurt+Peter Foundation continued its dialogue with Myonexus Therapeutics regarding manufacturing for a gene therapy clinical trial.
On the occasion of the annual JP Morgan biotechnology conference in San Francisco, the Kurt+Peter Foundation met with the leadership of Myonexus Therapeutics to further discuss how the foundation could support manufacturing for a gene therapy trial for LGMD2C.
Thanks to the generosity of our friends and family, the Kurt+Peter Foundation held a very successful fundraiser. Thank you to everyone that contributed to the success of a terrific event!
The San Francisco Business Times published a nice article about the Kurt+Peter Foundation here.
To build on its grant funding preclinical work for gene therapy for LGMD2C, and recognizing that manufacturing for a clinical trial for LGMD2C was a significant expense and potential source of delay, the Kurt+Peter Foundation began discussions with Myonexus Therapeutics [link] to see if the foundation could help fund a deposit with the contract manufacturer that would guarantee a position in line for manufacturing for a gene therapy clinical trial.
The U.S. Food and Drug Administration grants orphan drug status for the exon skipping technique developed by Dr. Elizabeth McNally, currently the Director of the Center for Genetic Medicine at, Northwestern University Feinberg School of Medicine.
Myonexus Therapeutics launches to develop and commercialize the technology developed at Nationwide Children’s Hospital for gene therapy for five different forms of limb girdle muscular dystrophy, including LGMD2C.
OptionB.Org, a foundation dedicated to helping individuals build resilience in the face of adversity, published the story of Kurt, Peter, Charlotte, and Scott, and the efforts of the Kurt+Peter Foundation.
The Kurt+Peter Foundation made a $126,000 grant to Nationwide Children’s Hospital to fund pre-clinical work necessary to pursue gene therapy for LGMD2C.
The Kurt+Peter Foundation made a $35,000 grant to Northwestern University to further the pre-clinical research into exon skipping being conducted by Dr. Elizabeth McNally.
P.O. Box 691
Menlo Park, CA 94026
Every donation brings us closer to a successful treatment and cure for Kurt, Peter, and other children around the world.