Developing effective AAV-based gene therapies is challenging, rewarding — and always a team effort. The work Viralgen does is one link in an impressive chain that leads to breakthrough treatments for rare, debilitating diseases.
We’re especially proud to be part of the consortium led by the Broad Institute that just received funding from the Advanced Research Projects Agency for Health (ARPA-H) THRIVE program to develop gene therapies for rare childhood diseases.
The award, for up to $34.5 million, will fund the Pediatric Epilepsies and Rare CNS (PERC) Gene Editing Platform — a collaboration of academic researchers, clinicians, patient advocates, and biotechnology companies — using the novel central nervous system-targeted AAV capsid, TfR1 CapX™, from Viralgen partner Apertura.
The reality is that making powerful tools like base editing and engineered capsids into real therapeutics requires building robust ecosystems of people and companies that share the same vision for the future of genetic medicines. Viralgen is excited to be a part of one of those ecosystems here.
As Andy Holt, Viralgen’s Chief Commercial Officer at Viralgen, put it, “The groups involved in THRIVE are so full of experts and passionate advocates that it is almost a little intimidating to be a part of. We're humbled to be able to contribute our experience making CapX and other CNS-directed vectors to bringing new therapies and new technologies to patients who desperately need better treatment options.”
Read more about this important AAV-based gene therapy project here.