Hey everyone, happy summer-time wishes from GP2C! I’m hoping this letter finds you well, and everyone has had a chance to wind down and enjoy a little summer fun. I thought I would dedicate this quick letter to a few of the research items we’ve been following here at GP2C!

Of course, we have spoken at length about the Avexis gene therapy studies, and the very positive news of the Novartis/AveXis acquisition. Progress continues with the recent announcement of plans to establish a new production facility for the development and manufacture of AVXS-101, -201 and -301 (with -201 being the “high interest therapy” to our community). Visit the link below for more information, but here’s a clip from the article:

AveXis is expanding its operations to North Carolina, opening up a new manufacturing center in Durham County, North Carolina Gov. Ray Cooper recently announced in a press release.

The company is anticipated to invest about $55 million into the new facility and open up about 200 new jobs, giving a boost to the state’s economy while improving its capabilities to manufacture therapies targeting rare neurological and genetic diseases… “Our primary focus is to bring gene therapies to patients suffering from devastating rare neurological diseases — such as SMA, genetic amyotrophic lateral sclerosis, and Rett syndrome — and continued investment in establishing our manufacturing infrastructure is a critical component to accomplishing this goal,” said Andrew Knudten, senior vice president of technical operations and chief technical officer of AveXis.

And how about this from our friends at RSRT… a recent award of over $900,000 to Dr. Stuart Cobb at the University of Edinburgh to pursue Spliceosome-Mediated RNA Trans-Splicing (SMaRT). Here’s a clip from that announcement:

Spliceosome-Mediated RNA Trans-Splicing (SMaRT). This technology allows a mutation to be spliced out and repaired in RNA, the intermediate between DNA and protein in the cell. The advantage is that this approach avoids any potential over-expression issues that could result from directly modifying DNA or giving extra protein. Dr. Cobb’s approach aims to correct mutations in the intermediate RNA transcript, allowing the MECP2 gene and the MeCP2 protein product to remain under normal gene expression controls...Once the SMaRT approach is optimized, it will be tested in mice with the full human MECP2 locus that contains the MeCP2 gene and all of its regulatory elements, to determine if this approach can be used in humans.

These are a just a couple of the latest announcements from RSRT research activities that we support through your generous donations! And finally, this from the RSRT Gene Therapy Consortium:

…The data generated by the Consortium exceeded our expectations. They were able to develop a gene therapy product candidate with impressive efficacy, safety and delivery characteristics. Importantly, the magnitude of improvement in the mouse models of Rett is much greater than that of any drug tested and suggests that significant benefit may be achieved in people. We expect improvements, at least to some degree, regardless of age.

As exciting as these and other announcements may be, we must stay focused on the day-to-day support and development of our Rett children. I know you do, because I hear positive stories (almost daily) of families and support teams fighting the battles to keep our kids strong and ready. This means never losing sight of the physical, emotional and educational needs of our Rett kids. We must win the battles and then win the war!

Exciting breakthroughs are coming – I believe this with all my heart and soul, and I hope you do also. GP2C is here to help, not only with our research targeted donations, but also through our family support teams and Rett University literacy programs. Let’s make sure our kids are ready!


Kevin, GP2C Director of Development