The event will feature presentations from Insmed's management team and scientific leaders, as well as from John Day, professor of Neurology, Pediatrics, and Pathology at Stanford University, and Michael Kelly, chief scientific officer of CureDuchenne.
"Our singular focus at Insmed has always been on patients – developing and delivering therapies that will make a transformational impact on their lives, regardless of modality or therapeutic area," said Will Lewis, chair and CEO of Insmed.
"Across our first three pillars, this has been the standard against which we have measured success, and we have proven our ability to deliver on our promise to patients at every stage of drug development and commercialization. Today, I look forward to unveiling the unique platforms and capabilities that will fuel our next phase of growth and, together with our first three pillars, put us on the path to become one of the next great, sustainable biotechnology companies.
“Driven by our highly skilled, industry-leading research teams, we believe the technologies we are progressing can address the challenges facing today's gene therapies and therapeutic proteins to deliver the next generation of first-in-class, first-in-disease, and potentially best-in-class medicines."
Gene Therapy with Targeted Delivery
Insmed has developed a targeted adeno-associated virus (AAV) delivery system to potentially address some of the key challenges associated with current gene therapies, including high dose requirements, inherent systemic toxicities, low efficacy, and off-target transduction.
This targeted mode of delivery has the potential to reduce the needed dose 10- to 50-fold, which may in turn offer an enhanced safety profile without limiting efficacy.
Key updates from this platform include: Insmed's first gene therapy candidate using a targeted intrathecal delivery approach will be in DMD, a devastating genetic disorder characterized by progressive muscle degeneration and weakness.
The company also announced a $500,000 equity investment from CureDuchenne Ventures, the investment arm of CureDuchenne, to support the development of Insmed's gene therapy program for DMD.
In addition, Insmed plans to initiate a gene therapy clinical trial in DMD later this year, with muscle biopsy data expected in the first half of 2024.
RNA End-Joining (REJ) Technology
Insmed said that its REJ technology has the potential to enable large-size gene delivery, overcoming the capacity challenges associated with traditional AAV vectors. This technology may make it possible to employ gene therapy for diseases caused by large genes that have historically not been viable targets for AAV gene therapy.