Cell therapy weekly: collaboration for solid tumor CAR-T therapy
This week: Dispatch Bio (PA, USA) and Bristol Myers Squibb (BMS; NY, USA) entered into a clinical supply and collaboration agreement to support the development of Dispatch’s novel solid tumor treatment, Ensoma (MA, USA) announced it will present preclinical data demonstrating proof-of-concept for its in vivo, HSC-derived CAR-M, NK and T cell platform, and Neurogene (NY, USA) dosed the first participant in its Rett syndrome gene therapy trial.
The news highlights:
- Collaboration for solid tumor CAR-T therapy
- In vivo HSC-derived CAR therapies
- First participant dosed with gene therapy for Rett syndrome
Collaboration for solid tumor CAR-T therapy
Dispatch Bio and BMS have entered into a clinical supply and collaboration agreement to support the development of Dispatch’s novel solid tumor treatment, DISP-10.
DISP-10 uses a two-step approach to treat solid tumors. First, DV-10, a tumor-specific virus, delivers a modified BCMA protein directly to cancer cells, tagging them for immune recognition. It also delivers IL-18 and CXCL-9 to reengineer the tumor microenvironment to be more favorable for T cells. Second, idecabtagene vicleucel (ide-cel), BMS’s BCMA-directed CAR-T therapy, recognizes and attacks the tagged tumor cells. This sequential process essentially converts solid tumors into targets that CAR-T cells can effectively eliminate, similar to how these treatments work against blood cancers.
Under the terms of the agreement, BMS will provide ide-cel for use in Dispatch’s first US Phase I trial, which is set to commence in 2026.
“Partnering with Bristol Myers Squibb – a global cell therapy leader and an early investor in Dispatch – speaks to the potential of our Flare platform and scientific approach,” said Naveen Bazaj, Senior Vice President, Corporate Development, at Dispatch. “This agreement represents the first time a company has secured a clinical supply for an autologous CAR T-cell therapy and positions us to accelerate development of our DISP-10 program, which we plan to enter the clinic in 2026.”
In vivo HSC-derived CAR therapies
Ensoma, an in vivo hematopoietic stem cell (HSC) engineering company, will present new preclinical data demonstrating proof-of-concept for its in vivo, HSC-derived CAR-M, NK and T cell platform at the Society for Immunotherapy of Cancer (SITC) 40th Annual Meeting (5-9 November 2025; National Harbor; MD, USA).
“While ex vivo CAR-T therapies have transformed treatment for blood cancers, use in solid tumors has been limited by multiple factors, including poor T cell infiltration and persistence in the immunosuppressive tumor microenvironment, as well as manufacturing cost and complexity,” said Jim Burns, CEO of Ensoma. “By engineering HSCs in vivo, we can develop off-the-shelf therapies that turn the body into its own cell factory—capable of continuously producing multiple CAR immune cell types that work together against solid tumors. These data move us closer to realizing this vision as we advance toward our first in vivo, HSC-derived CAR-M, NK, and T development candidate early next year.”
Presentations:
Title: Discovery of lineage specific regulatory elements for development of in vivo CAR immune cell therapy via hematopoietic stem cell engineering
Abstract Number: 1019
Poster Presentation Time/Date: 7 November 7, 5:10-6:35 pm EST
Title: In vivo HSC engineering with Ensoma’s virus like particles (VLPs) generates lineage-restricted, multiplexed CAR-M, NK, and T cells to cooperatively mediate solid tumor control in pre-clinical models
Abstract Number: 302
Poster Presentation Time/Date: November 8, 5:10-6:35 pm EST
First participant dosed with gene therapy for Rett syndrome
Neurogene announced that the first participant has been dosed in the Embolden™ registrational clinical trial investigating the company’s gene therapy for Rett syndrome, NGN-401.
“Dosing the first participant in the Embolden registrational trial of NGN-401 marks a major milestone in advancing a gene therapy for Rett syndrome. We prioritized operational excellence this year, doubling our US site footprint and initiating 12 of 13 sites to meet patient demand, positioning us to complete enrollment within the next three to six months,” said Rachel McMinn, Founder and EO of Neurogene. “The Embolden trial is a single registrational trial designed to evaluate NGN-401 in females ages three and above, generating key insights into its potential when administered early in the disease course and supporting a broad label strategy across a wide age range.”
The treatment involves a single injection directly into the brain’s cerebrospinal fluid system, delivering a specific dose designed to restore the missing MECP2 protein that causes Rett syndrome. Preclinical studies showed this delivery method produced better therapeutic gene expression in critical brain areas compared to spinal injection approaches. The trial is currently recruiting patients across the United States, with enrollment expected to finish within three to six months.