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.
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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.
United StatesFull profile →
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.
BelgiumFull profile →
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.
United StatesFull profile →
Develops circular RNA (oRNA) and LNP delivery. Its current LinkedIn page identifies it as a wholly owned Eli Lilly subsidiary, so it should not be treated as an independent private platform company.
United StatesFull profile →
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.
United StatesFull profile →
An RNA and in vivo immune reprogramming platform developer; the website identifies it as a Bristol Myers Squibb company.
United StatesFull profile →
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.
United StatesFull profile →
STX-001 is an intratumoral self-replicating mRNA treatment encoding IL-12. The company discloses a Phase 1 study and a broader programmable RNA platform.
United StatesFull profile →
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.
United StatesFull profile →
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.
United StatesFull profile →
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.
United StatesFull profile →
Kernal combines T-cell-targeted LNP delivery with selective mRNA translation for in vivo CAR-T. Its ASGCT 2026 announcement concerns preclinical programming studies. An ARPA-H award supports development; it is research funding, not clinical validation or a venture financing round.
United StatesFull profile →
EXUMA’s GCAR platform combines CD3-targeted lentiviral delivery with FITNESS DRIVER for in vivo CAR engineering. Its separate rPOC SC platform incubates PBMCs with vector outside the body and should not be described as fully in vivo manufacturing. Current GCAR clinical stage was not verified.
United StatesFull profile →
TPST-4003 combines CD7-targeted mRNA/LNP delivery with a CD19/BCMA CAR; first IIT dosing was projected for Q4 2026, not verified as completed. Tempest separately obtained an option to license Senlang’s clinical lentiviral platform. TPST-2003 clinical results concern ex vivo CAR-T.
United StatesFull profile →
Senlang’s CD7-targeted lentiviral in vivo CAR-T portfolio includes a BCMA/GPRC5D candidate in Phase 1 dose escalation for relapsed/refractory myeloma. Partner Tempest disclosed an exclusive option in September 2026; an option is not an exercised license or acquisition.
ChinaFull profile →
CREATE, formerly Myeloid Therapeutics, disclosed preclinical in vivo CAR-T/CAR-NK and all-RNA RetroT work at AACR 2026. Clinical MT-302 is a myeloid-cell program, not evidence that its CAR-T programs have reached the same stage. The former name is retained as an alias.
United StatesFull profile →
Vyriad develops targeted viral genetic medicines and oncolytic therapies. Its BCMA-directed in vivo CAR-T candidate VV169 uses a retargeted lentiviral vector to engineer T cells inside the body. On September 10, 2026, the company announced that its Phase 1 study in relapsed/refractory multiple myeloma was open for enrollment at Mayo Clinic following FDA IND clearance. Patient dosing and clinical benefit were not established in that announcement.
United StatesFull profile →
CPTx develops single-stranded DNA payloads with nonviral LNP delivery for in vivo CAR-T. The company reports small-animal validation; durability, redosing, and immune-silence statements remain company claims. CPTx is a distinct company from Capstan and the CPTX2309 drug code.
GermanyFull profile →
LB2501 is a CD19/CD20 in vivo CAR-T candidate using the TaVec lentiviral platform, with an ongoing China Phase 1 study (NCT07002112). Its October 2026 U.S. IND clearance supports a planned Phase 1/2 study. Neither IND clearance nor early responses constitute approval.
United StatesFull profile →
IASO reported first-in-human Phase 1 data for BCMA-directed IASO206 in relapsed/refractory myeloma at IMS 2026. It also partners with Umoja on CD22 programs with territorial rights. IASO206 and Umoja’s CD22 UB-VV400 are different programs.
ChinaFull profile →
Founded in 2019, the company develops mRNA, self-replicating RNA, and delivery platforms. In 2026 it reported early JCXH-213 results in two patients with ITP; these findings should be distinguished from formal registered-trial phases and independent validation.
ChinaFull profile →
GRIT disclosed T-cell-targeted mRNA/LNP GT801 and a separate lentiviral direction. GT801 is also developed through spinout Vivacta Bio; the two company profiles describe a shared program lineage, not two independent drugs. GRIT’s TIL clinical stages are separate.
ChinaFull profile →
Genocury’s September 2025 announcement reports an investigator-initiated study of CD19-directed in vivo CAR-T in B-cell lymphoma. A reported individual response is preliminary and does not establish durability or comparative benefit. Current enrollment and formal regulatory trial status need confirmation.
ChinaFull profile →
Coverage expands from sourced records; this database does not claim all biopharma companies.