RARE Daily

Insmed Boasts Platforms, Six INDs by End of 2025, and New Investment

May 10, 2023

Rare Daily Staff

Insmed said it raised $500,000 through an equity investment from CureDuchenne Ventures to support the development of a gene therapy for the rare neuromuscular disease Duchenne muscular dystrophy and said it expects to file six INDs by the end of 2025.

The news came with a string of other announcements touting the company’s progress ahead of an event for analysts and investors.

“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,” said Will Lewis, chairman and CEO of Insmed. “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.”

Insmed said it 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.

The company plans to initiate a gene therapy clinical trial in Duchenne muscular dystrophy (DMD) later this year, with muscle biopsy data expected in the first half of 2024. The company said it has identified additional gene therapy targets and it has begun preclinical work on these.

“While exciting advances have led to clinical trials of gene replacement approaches for DMD, the current landscape is complicated by requiring high doses of viral vectors that raise safety and tolerability concerns, resulting in significant debate and lack of clarity on how best to manage patients with this devastating disease,” said John Day, professor of neurology, pediatrics, and pathology at Stanford University. “The possibility of a more targeted, lower-dose therapy that could reduce systemic toxicity and provide a safer option for patients is very promising.”

Insmed also highlighted its RNA-End-Joining (REJ) technology. It said REJ 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.

The company plans to target Stargardt disease, a genetic retinal disease that causes vision loss typically beginning in childhood, with its first REJ gene therapy candidate. Patients with Stargardt disease currently have no approved therapies to treat their condition. The company said it expects to submit an IND for the treatment of Stargardt disease by the end of 2024.

Insmed is also exploring the potential of REJ to deliver larger dystrophin protein in DMD.

Insmed’s also discussed its proprietary protein deimmunization platform, Deimmunized by Design (DbD), which it said offers a potential solution to the immunogenicity challenges associated with both gene therapies and protein therapeutics by using artificial intelligence to reengineer proteins to silence their immunogenic properties while maintaining their therapeutic activity.

Using this platform, Insmed is deimmunizing several therapeutic proteins and viral vectors, including AAV capsids, which could unlock the potential for redosable gene therapy. It is exploring the potential of redosable gene therapy using a deimmunized AAV capsid in the treatment of argininosuccinic aciduria (ASA), a rare, urea cycle disorder that typically presents in newborns, and expects to have preclinical data in 2024.

Finally, the company said it is advancing a proprietary manufacturing technique that leverages the unique properties of algae to potentially reduce the time, cost, and complexity of producing therapeutic proteins and viral vectors for gene therapy. This next-generation approach may offer potential benefits including rapid scalability, no plasmid transfection, low production costs, and ease of culturing and maintenance.

Insmed expects to advance to full AAV capsid production using this manufacturing platform in 2024.

Photo: Will Lewis, chairman and CEO of Insmed

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