Cellectis pivots away from allogeneic CAR-T to in-vivo gene editing
Cellectis is ending development of its two clinical-stage allogeneic CAR-T programmes, lasme-cel and eti-cel, redirecting the company toward in vivo gene editing. The French biotech had been due to read out a pivotal Phase 2 trial of lasme-cel in B-cell acute lymphoblastic leukaemia by year-end, alongside a full Phase 1 dataset for eti-cel in non-Hodgkin lymphoma. The decision follows changes in the competitive landscape for B-cell acute lymphoblastic leukaemia (B-ALL) and non-Hodgkin lymphoma (NHL), where Cellectis said advances in frontline treatments have reduced the number of patients progressing to later lines of therapy.
The company is now channelling resources into two preclinical programmes, HEAL-101 (in vivo base editing, targeting APOC3 for severe hypertriglyceridemia) and HEAL-201 (in vivo epigenetic editing targeting PCSK9 for severe hypercholesterolemia), with plans to move both into Phase 1 studies in China. Preliminary readouts are expected in the second half of 2027 and first half of 2028 respectively.
The shift adds to the broader industry movement towards in vivo therapies, including Kite Pharma walking away from a $2.3 billion allogeneic deal with Shoreline Biosciences in favour of in vivo acquisitions, alongside big-ticket in vivo buys from AbbVie, Bristol Myers Squibb, AstraZeneca and Eli Lilly.
Read more via Biopharma Dive
FDA aligns with Rocket Pharmaceuticals to continue pivotal RP-A501 trial
Rocket Pharmaceuticals has reached agreement with the FDA on the continued enrolment and dosing of its pivotal Phase 2 trial of RP-A501 for Danon disease, following the successful treatment and safety observation of the first three patients under a modified protocol.
The FDA has confirmed a pivotal efficacy population of 12 patients, including the first three patients treated under the modified protocol. Following review of the initial safety data, the agency has supported continued enrolment and dosing using the recalibrated dose and refined immunomodulatory regimen. The development follows a clinical hold placed on the programme in 2025. The FDA lifted that hold in August 2025, allowing Rocket to resume the study initially with three patients at a recalibrated dose of 3.8 × 10¹³ GC/kg.
Rocket reported in August that the first three patients treated under the modified Phase 2 protocol had completed the required safety observation period without thrombotic microangiopathy, capillary leak syndrome or other significant safety concerns. The FDA has now confirmed the established 12-month co-primary endpoints, which Rocket intends to use to support a potential accelerated approval pathway.
Read more via Clinical Trial Vanguard
Ride Therapeutics to receive up to $7.3 million from Cystic Fibrosis Foundation for delivery technology
Ride Therapeutics has secured a strategic investment of up to $7.3 million from the Cystic Fibrosis Foundation to advance its Molecular Logistics platform and develop next-generation genetic medicine delivery technologies for cystic fibrosis.
The platform builds ultra-diverse libraries of millions of non-viral nanoparticle carriers, evaluating them in vivo following systemic or inhaled administration to determine which tissues and cell types each carrier reaches. The resulting data trains a generative AI design engine that produces optimised carriers for specific tissues. The new funding will accelerate Ride's existing work on IV delivery and support expansion into inhaled delivery. Ride was spun out of Harvard University and the University of Cambridge in 2023 on an $8 million seed round.
The funding highlights the increasing focus on delivery as a critical enabling technology for the next generation of genetic medicines. While many therapeutic platforms have advanced rapidly, achieving efficient and tissue-specific delivery remains a major challenge, particularly for organs and cell types outside the liver.
Read more via Cystic Fibrosis News Today
Sensorion confidentially files for potential US IPO
French hearing-loss biotech Sensorion has taken the first formal step towards a potential US listing, confidentially submitting a draft Form F-1 registration statement to the US Securities and Exchange Commission as it advances its lead gene therapy programme targeting GJB2-related hearing loss. The filing covers a potential offering of ordinary shares, including American Depositary Shares, on a US national exchange.
The company, which is already listed on Euronext Growth in Paris, has not disclosed the potential size, timing or terms of the offering. Sensorion stressed that any IPO would remain subject to market and other conditions.
A potential US listing would give Sensorion an additional route to the US capital markets as it moves its lead gene therapy programme into clinical development.
Read more via European Biotechnology
RegCell raises $66 million to advance immune tolerance platform into the clinic
RegCell has closed a $66 million funding round, combining a fully subscribed $44 million Series A with a $22 million non-dilutive grant from Japan's AMED, to move its epigenetic reprogramming platform into clinical-stage development across multiple autoimmune indications.
The company's approach builds on the discovery of regulatory T cells (Tregs) by RegCell scientific co-founder Shimon Sakaguchi, whose research contributed to the understanding of how Tregs maintain immune tolerance and was recognised with the 2025 Nobel Prize in Physiology or Medicine. RegCell is developing an epigenetic reprogramming platform designed to convert disease-driving CD4+ T cells into stable, antigen-specific Tregs while preserving their native T-cell receptor specificity. This approach could offer a more targeted alternative to broad immune suppression by restoring immune tolerance rather than simply suppressing immune activity. Its platform is being developed across multiple autoimmune indications, with the new financing expected to support an IND submission, generate human proof-of-concept data and expand the clinical pipeline.
Read more via FirstWord Pharma
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