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Monica’s Perspective on the Novartis News

Yesterday was a tough day. The termination of the Novartis Rett gene replacement program, OAV-201, was a blow. For many of us, myself included, this program was the first tangible prospect for a better future for our children. This was a program that got its start in RSRT’s Gene Therapy Consortium. So it was hard both personally and professionally to see the program come to end.

I start most of my mornings by reviewing the headlines from the biopharmaceutical industry. Every single day I read about disease programs being terminated, sometimes due to science reasons, other times for business reasons. It’s easy to read about these comings and goings when the decision doesn’t affect your daughter, your community.

While the disappointment was great, I can’t say the surprise was. The lack of updates from Novartis was worrisome. Despite assurances from Novartis that community updates would be quarterly the last one was in February 2021.

The silver lining, however, is that RSRT has always mitigated both science and business risk by pursuing multiple partnerships with companies and treatment strategies in parallel.

The Novartis program wasn’t the only gene replacement effort in our portfolio. Our Gene Therapy Consortium and MECP2 Consortium also laid the foundation for Taysha Gene Therapies’ gene replacement program, which is on track, pending FDA approval, to start soon.

Gene replacement is just one of six strategies RSRT is pursuing now, and within each strategy there are multiple programs. This diversification of strategies and partnerships protects against setbacks like this one.

In addition, there are other biopharmaceutical companies that have not yet publicly disclosed their Rett programs. We are looking forward to seeing what comes from our DNA and RNA editing partner companies, as well as companies focused on MECP2 reactivation. Truly, we have a lot to look forward to!

So while I experienced a myriad of emotions yesterday, they did not include resignation or helplessness. While the day may have started with frustration and disappointment, it ended with resolute determination. No one at RSRT will rest until there is a cure.