Massachusetts-based biotech company Generation Bio has announced a massive restructuring, cutting 90% of its staff as it struggles to secure funding for its cell-targeted lipid nanoparticle (ctLNP) technology, a potential breakthrough in autoimmune disease treatment.
Generation Bio’s platform focuses on liver-related and immune cell delivery using ctLNPs. One of its key innovations is a T-cell-specific ligand, enabling targeted delivery of small interfering RNA (siRNA) to T cells for autoimmune disease therapy.
In a recent preclinical nonhuman primate study, a 0.5 mg/kg dose of ctLNP-siRNA achieved a “significant knockdown” of beta-2 microglobulin—a reporter protein—in T cells over three weeks. These results build on prior research showing ctLNP’s efficient and selective delivery of various therapeutic payloads, including mRNA and the company’s proprietary immune-quiet DNA.
Despite promising early-stage data, Generation Bio says it faces a shortage of two critical resources: time and capital.
“Our delivery system is mature, but our program data are early,” said CEO Geoff McDonough. “We understand the investment and time needed to demonstrate proof-of-concept in patients, and the uncertainty of achieving that within our current cash runway.”
The company is now reviewing strategic alternatives to maximize value for shareholders and potentially advance its pipeline through partnerships or asset sales.
As part of its restructuring—expected to be completed by the end of October—Generation Bio will maintain a smaller core R&D team while extending its $141.4 million cash balance “into the foreseeable future.”
Founded with a vision to deliver gene therapies for liver and eye diseases, Generation Bio went public on Nasdaq in 2020. Following a setback in its hemophilia A program in 2021, the biotech shifted focus in 2023 to non-liver targets, which led to a prior 40% workforce reduction, including the departure of its chief medical and chief development officers.
The latest layoffs mark the company’s most dramatic downsizing yet, underscoring the funding hurdles facing biotech innovators—even those with potentially groundbreaking drug delivery platforms.
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