Generation Bio provides a cell‑targeted lipid nanoparticle (ctLNP) platform that delivers siRNA specifically to T cells in vivo, enabling sequence‑specific knockdown of disease‑relevant genes. The stealth formulation minimizes off‑target organ uptake and supports repeat dosing with predictable exposure, offering a modular solution for pharmaceutical and biotech developers of T‑cell‑driven autoimmune therapies.
Funding
$76M raised to dateRaised to date based on public sources. This may differ from the amount the company actually raised and is based only on what is publicly available on the internet.
MFounders
Product
Problem
Autoimmune diseases driven by autoreactive T cells cause chronic tissue inflammation and organ damage. Existing therapies broadly suppress the immune system, leading to limited efficacy and significant side effects. There is no clinically viable method to selectively modulate pathogenic T‑cell activity at the genetic level.
Solution
Generation Bio has engineered a cell‑targeted lipid nanoparticle (ctLNP) platform that delivers short‑interfering RNA (siRNA) directly into T cells in vivo. The ctLNP incorporates a ligand that binds a T‑cell‑specific surface receptor, while a stealth lipid composition minimizes opsonization and reduces liver and spleen clearance to under 1 %. Once internalized, the nanoparticle releases siRNA into the cytoplasm, enabling potent, sequence‑specific knockdown of disease‑relevant genes. This approach provides genetic precision and tunable pharmacology, allowing repeat dosing with predictable exposure. By sparing other immune cells, the therapy aims to achieve higher potency and better tolerability than conventional immunosuppressants. The platform is modular, permitting rapid re‑engineering of the targeting ligand and siRNA payload to address multiple undruggable T‑cell targets across a range of autoimmune indications.
Target Audience
The primary customers are pharmaceutical and biotech companies developing treatments for T‑cell‑driven autoimmune diseases, as well as academic and contract research organizations seeking a selective in‑vivo gene‑silencing tool for T‑cell biology.
Features
- Ligand‑decorated lipid nanoparticle that selectively binds and internalizes into T cells via a defined surface receptor.
- Stealth formulation that evades serum protein binding and reduces hepatic and splenic uptake to <1 % of injected dose.
- siRNA payload delivery achieving efficient cytoplasmic release for robust gene silencing in T cells.
- Modular architecture allowing interchangeable ligands and siRNA sequences to target new intracellular proteins.
- Redosable dosing regimen with consistent pharmacokinetics and a wide therapeutic index.
- Compatibility with a broad library of chemically modified siRNAs to enhance stability and potency.
- Scalable manufacturing process based on established lipid nanoparticle production technologies.