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Disease tags identify mentions in recorded facts or pipeline text, not approved indications or a complete pipeline. Platform service providers may have no disease-specific tag.
67 matching companies
Aanastra discloses PEP-CAR, an anti-CD19 in vivo CAR-T strategy combining engineered RNA and targeting peptides for cancer and autoimmune disease. The public website does not establish a registered clinical program or validated patient outcomes.
AAVivo lists CD19 AVO-100, BCMA AVO-101, and dual BCMA/CD19 AVO-102 using T-cell-targeted AAV. The disclosed activities include vector production and construct optimization, not verified clinical dosing. AVO-103 is a TCR program and is kept separate from CAR-T.
AERA-109 is a targeted-LNP in vivo CAR-T candidate for B-cell-mediated autoimmune diseases, supported by mouse and nonhuman-primate studies. The pipeline projects clinical entry in the second half of 2026; a projected date is not proof that dosing has begun.
Alaya is a preclinical in vivo CAR-T developer. Its disclosed polymeric nanoparticle platform encapsulates lentiviral CAR vectors for targeted T-cell transduction; this is a hybrid polymer/viral approach, not simply a nonviral polymer delivery system.
AllerGene proposes in vivo-delivered mRNA CAR-T to target mast cells in allergic disease. Its website establishes research intent, but does not establish a named clinical candidate, patient dosing, or validated allergy outcomes. It is distinct from Allogene Therapeutics.
AlphaNa identifies AFN50 as an mRNA-based in vivo CAR-T program for autoimmune disease currently evaluated in IITs. The disclosed program is classified as investigator-initiated research; unrelated RNA programs and inconsistent codes elsewhere on the website are not used to infer additional CAR-T assets.
Intellia presents CRISPR gene-editing science, clinical programs, and scientific publications on its official website. Its gene-editing work belongs in this broader in vivo engineering field; it should not be labeled as equivalent to in vivo CAR-T.
Beam develops investigational precision genetic medicines using base editing. Its website describes a delivery strategy spanning electroporation and nonviral and viral approaches. Those routes include different applications; the platform label alone does not establish in vivo efficacy.
AbbVie completed its Capstan acquisition in August 2025. CPTX2309 uses targeted LNP in vivo CAR-T technology; the sponsor lists NCT06917742 as a recruiting Phase 1 study in healthy volunteers, rheumatoid arthritis, and SLE. Registration does not establish efficacy.
Tessera develops Gene Writing and RNA delivery technologies for genomic changes and targeted in vivo applications. Its November 2026 ASH announcement concerns preclinical studies, including CAR-T generation in nonhuman primates.
Tune develops TEMPO epigenetic regulation to activate or silence gene expression without cutting or changing the DNA sequence. Epigenetic modulation should be distinguished from sequence editing and permanent DNA insertion.
Sana develops CD8-targeted fusosome in vivo CAR-T, including SG293 and BCMA-directed SG227. Its August 2026 update describes SG293 clinical-start preparations and surrogate nonhuman-primate data, with SG227 clinical entry projected as early as 2027. Human dosing was not verified in this review.
Precision BioSciences develops ARCUS-based in vivo editing for gene elimination, excision, and insertion. Delivery and evidence differ by program; the company describes AAV delivery for its DMD gene-excision approach.
VivoVec uses engineered lentiviral vectors for in vivo CAR-T. The disclosed pipeline includes CD22 UB-VV400/410 and BCMA×GPRC5D UB-VV500. IASO retains Greater China rights to the partnered CD22 programs; UB-VV111 is partnered with AbbVie. Trial registration and IND clearance do not establish efficacy.
AstraZeneca completed its acquisition in May 2025. ENaBL is a targeted lentiviral platform; anti-BCMA ESO-T01 has a published Phase 1 study in multiple myeloma. The small, uncontrolled study provides preliminary evidence, not established benefit.
KLN-1010 is a BCMA-directed in vivo CAR-T candidate in Phase 1 for multiple myeloma, using the lentiviral iGPS platform. Lilly confirmed completion of the Kelonia acquisition in its Q2 2026 results. Early clinical findings remain investigational.
Orna develops circular RNA in vivo cell therapies, including CD19-directed ORN-252 for B-cell-driven autoimmune diseases. Lilly completed the acquisition in 2026; its Q3 filing identifies ORN-252 as Phase 1 at acquisition. ReNAgade was acquired by Orna in 2024 and is not counted separately.
Interius develops targeted lentiviral in vivo CAR therapies. Its clinical lead INT2104 targets CD20 and engineers T and NK cells. Gilead’s annual filing confirms that the acquisition closed in October 2025; Interius is a wholly owned subsidiary.
BMS completed its acquisition in December 2025, obtaining OTX-201, a preclinical RNA-based in vivo CAR-T program for autoimmune disease. The filing establishes ownership and the disclosed development stage; it does not report human efficacy.
Sail combines eRNA with targeted nanoparticles for in vivo CAR-T in immune-mediated diseases. Its July 2026 Johnson & Johnson collaboration includes an option to acquire Sail, not a completed acquisition. A named clinical CAR-T candidate is not established by this announcement.
The pipeline identifies STX-005 as a circular mRNA circuit for in vivo CAR-T in autoimmune disease and blood cancers. Clinical findings from the separate IL-12 programs STX-001/003 must not be attributed to STX-005; current CAR-T clinical initiation was not verified.
Azalea develops enveloped delivery vehicles (EDVs) for targeted in vivo T-cell genome engineering. Its disclosed preclinical approach includes precise TRAC-locus CAR insertion. This combines delivery and site-specific integration; human therapeutic benefit has not been established.
Ensoma disclosed mouse proof of concept for in vivo HSC engineering that generates CAR-bearing macrophage, NK, and T-cell lineages. This is an HSC-derived, multi-lineage approach, distinct from directly transducing mature T cells. CAR-T human efficacy is not established.
NanoCell’s NanoCAR approach uses targeted LNPs to generate CAR-T cells in vivo. Its animal proof-of-concept disclosure describes nonviral DNA delivery; this should not be classified as a transient mRNA-only platform. Clinical efficacy remains unestablished.
Coverage expands from sourced records; this database does not claim all biopharma companies.
