Cell therapy weekly: FDA draft guidance to help accelerate cell and gene therapies
This week: the US Food and Drug Administration (FDA; MD, USA) issued a draft guidance aiming to accelerate cell and gene therapies and a UK-based biotech company was awarded US$2.8 million in grants to support the development of its gene-control platform. Plus, Legend Biotech (NJ, USA) presented clinical data on two of its CAR-T therapies at the American Society of Clinical Oncology Annual Meeting (ASCO; May 26–June 2, 2026; IL, USA).
The news highlights:
- US$2.8 million grant to support gene-control platform for advanced therapies
- Legend Biotech presented encouraging CAR-T pipeline data at ASCO
- FDA draft guidance to help accelerate cell and gene therapies
US$2.8 million grant to support gene-control platform for advanced therapies
Biotechnology company Laverock Therapeutics (London, UK) has been awarded two new grants worth approximately US$2.8 million to support the development of the company’s gene-control platform. The platform spans a wide range of therapeutic applications and cell types, including developing programmed T cells and macrophages for solid tumor indications and hypoimmunogenic pancreatic islet cells for type 1 diabetes, with pipeline expansion into additional non-oncology areas, building on previous awards in non-dilutive funding.
The first grant will scale the platform within T-cell products, using solid tumor patient datasets and data on intracellular signaling and antigen expression. The second grant will expand macrophage-based programs into non-oncology indications, leveraging the platform’s ability to program myeloid cell phenotypes and control therapeutic payload expression.
“Success in these two highly competitive grant competitions provides further validation of our approach and will enable us to expand our efforts across platform and product development,” expressed David Venables, CEO of Laverock therapeutics. “As we push towards the clinic for our lead program this additional funding will help unlock the true breadth of what our technology can achieve. We can’t wait to get started!”
Legend Biotech presented encouraging CAR-T pipeline data at ASCO
At the ASCO Annual Meeting 2026, Legend Biotech presented first-in-human clinical data for its DLL3-targeted CAR-T therapy, LB2102, for relapsed/refractory small cell lung cancer or large-cell neuroendocrine carcinoma. LB2102 demonstrated a manageable safety profile and encouraging clinical activity in solid tumors among heavily pretreated patients.
“Patients with relapsed or refractory [small cell lung cancer] or [large-cell neuroendocrine carcinoma] have historically had very limited treatment options and poor outcomes,” said Zhonglin Hao, Professor of Medicine and Cancer Biology, Director, Thoracic Oncology Program, University of Kentucky Markey Cancer Center (KY, USA). “Durable disease control and responses at higher dose levels with LB2102 are encouraging, particularly alongside a manageable safety profile. These findings support continued evaluation of CAR-T approaches in solid tumors, where effective therapies remain limited.”
LB2102 wasn’t the only CAR-T therapy Legend Biotech reported positive data on at ASCO. New analyses from the CARTITUDE program demonstrated continued sustained clinical benefits and a consistent safety profile of CARVYKTI® in patients with multiple myeloma. Progression-free survival and overall survival benefits were observed across both high-risk and standard-risk cytogenetic populations among patients who responded to bridging therapy, with 30-month OS rates greater than 85%.
FDA draft guidance to help accelerate cell and gene therapies
The FDA issued draft guidance to help developers bring promising gene therapies to patients more efficiently. When finalized, the guidance will outline how sponsors can use publicly available information and established platform knowledge, including chemistry, manufacturing and controls (CMC) data, nonclinical study results and clinical information, to streamline regulatory submissions for human gene therapy products that use genome editing in human somatic cells.
“Today’s action reflects the FDA’s commitment to get safe and effective cell and gene therapies to patients faster, particularly those living with rare and life-threatening diseases who have few or no other treatment options,” said Karim Mikhail, Acting Director of the Center for Biologics Evaluation and Research. “By providing information on how companies may build on what is already known we are accelerating innovation without compromising the rigorous scientific standards that patients and the public depend on. Ultimately, this is about making sure that the promise of gene therapy reaches the patients who need it most, as quickly and safely as possible.”
