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Generation Bio to Use ctLNP for siRNA Autoimmune Therapies

Generation Bio

Novel programs will combine validated cell-targeted LNP (ctLNP) delivery with siRNA to selectively modulate T cells in vivo

Generation Bio Co.  a biotechnology company working to change what is possible for people living with T cell-driven autoimmune diseases, announced it is leveraging its validated T cell-directed lipid nanoparticle (ctLNP) to develop siRNA therapeutics to silence disease-driving targets in T cells.

“We are excited to move Generation Bio toward the clinic by deploying our ctLNP to deliver siRNA to T cells. By precisely modulating T cell activity in vivo we believe we can address high-value, currently undruggable targets involved in the inflammation and tissue damage associated with T cell-driven autoimmune diseases,” said Geoff McDonough, M.D., chief executive officer of Generation Bio. “Our aim is to silence therapeutic T cell targets without impacting other immune cell types, unlocking a powerful new application for siRNA in the field. We plan to submit our first IND in the second half of 2026 and to enter the clinic within our cash runway, which is into the second half of 2027.”

siRNA delivery to T cells has historically been limited by the inability to achieve selective cell targeting combined with efficient access to the cytoplasm where siRNA operates, challenges that Generation Bio has designed its ctLNP to overcome. The company recently presented non-human primate data demonstrating that its ctLNPs work by targeting T cells through a target receptor of interest with a strong selectivity for CD8+ and CD4+ effector T cells and NK cells, all of which are involved in auto-reactive tissue damage in a number of autoimmune diseases.

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siRNA delivered with ctLNP aims to modulate T cell function without impacting the function of the broader immune system. ctLNP-siRNA could reach targets that change how auto-reactive T cells activate, differentiate, migrate, and damage tissues. Generation Bio will provide further details about its lead ctLNP-siRNA programs in upcoming quarters and expects to submit its first IND in the second half of 2026. The company is reorganizing to support the clinical development of T cell-directed medicines.

In addition, the company is announcing changes to its executive leadership team. Phillip Samayoa, Ph.D., chief strategy officer, will succeed Matthew Stanton, Ph.D. as chief scientific officer. Dr. Stanton will remain with the company through mid-2025 and transition to its Scientific Advisory Board thereafter. Kevin Conway, the company’s head of finance, has been elected chief financial officer to succeed Matthew Norkunas, M.D. Generation Bio also expects to appoint a chief medical officer in 2025.

“I am grateful for the passion and expertise each person at Generation Bio has contributed to enable our transition toward the clinic,” said Dr. McDonough. “Matt Stanton has been instrumental in the invention of our technologies, and I look forward to our continued work together. Likewise, Matt Norkunas has built a terrific team here, which Kevin will continue to build upon. With a strong foundation in place, we are well-positioned to execute on our mission to bring highly differentiated T cell-directed therapies to patients.”

Source: GlobeNewsWire