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LVV-based Fully-human 4th Generation In vivo Dual-targeting CAR Platform

In vivo CAR Platform (LVV Route) leverages engineered modifications to the lentiviral vector's capsid and glycoprotein. It overcomes the technical limitation of conventional lentiviruses, which can only transduce activated T cells. This innovative dual-targeted LVV enables direct in vivo delivery of fully human, dual-target, 4th-generation CAR genes into T cells.

The platform has engineered and validated multiple novel glycoproteins, breaking the constraints of underlying VSV-G patents, with related patent applications filed. Going forward, tissue-specific promoters and B-cell-specific expression suppressor elements will be incorporated to further improve delivery precision and safety, offering patients a novel CAR-T therapeutic modality that requires no ex vivo cell manufacturing.

Core Advantages
  • Engineered lentiviral capsid/glycoprotein to overcome conventional transduction limitations
  • Bypasses foundational VSV-G patents with independent intellectual property portfolio
  • CD3/CD7 dual-targeted LVV, resolving the conflict between CD3 single-target tropism and T-cell activation-induced exhaustion
  • CAR construct adopts fully-human, dual-target, 4th-generation CAR design
  • Self-inactivating design reduces the risk of systemic T-cell activation by free circulating virus in vivo
  • T-cell-specific promoter enhances delivery precision and mitigates off-target risk
Pipeline Application
Skye-03

Skye-03 (BCMA/GPRC5D/FCRH5 dual-targeting, Multiple Myeloma)

cssDNA-LNP based Fully-human 4th Generation In vivo Dual-targeting CAR Platform

The In vivo CAR Platform (LNP Route) employs an innovative CD3/CD7 dual-targeted tLNP strategy (targeted lipid nanoparticles carrying circular single-stranded DNA) to achieve site-specific integration and delivery of fully human, dual-target, 4th-generation CAR genes into T cells in vivo.

Built upon proprietary engineered CAR scaffold sequences and original T-cell metabolic reprogramming regulation technology, this platform directly addresses key bottlenecks of current in vivo CAR-T therapies: rapid CAR-T cell exhaustion and short in vivo persistence. By optimizing T-cell functional status via metabolic reprogramming, the platform is expected to markedly enhance anti-exhaustion capacity and long-term persistence of CAR-T cells generated in the body.

Core Advantages
  • Innovative CD3/CD7 dual-targeted tLNP delivery strategy
  • Unique cssDNA-LNP for site-specific integration and delivery
  • Fully human, dual-target, 4th-generation CAR scaffold sequences
  • Original T-cell metabolic reprogramming technology to resolve in vivo CAR-T exhaustion and persistence limitations
Pipeline Application
Skye-04

Skye-04 (CD19/CD20/BAFF-R dual-targeting, B-NHL)

Fully-human Dual targeting Universal CAR Platform

The Universal CAR Platform is designed for autoimmune diseases, employing an allogeneic CAR-T (UCAR-T) strategy derived from healthy donors to overcome the limitations of autologous CAR-T in capacity, cost, and patient eligibility.

The platform leverages dual-target CAR structure optimization to boost target cell recognition and elimination. Combined with CRISPR-Cas9 gene editing of donor T cells, it reduces the risk of graft-versus-host disease (GVHD) and extends the in vivo persistence of UCAR-T cells. A single batch can yield product for 500–1000 patients, with manufacturing cost only 1/10 to 1/30 of autologous CAR-T. It delivers an accessible cellular immunotherapy option for patients with autoimmune disorders.

Its lead product features a CD7/BCMA dual-target design. For the first time, it breaks the constraint that CD19, BCMA single or dual-target CAR-Ts can only treat a subset of autoimmune diseases driven by autoantibodies from B and plasma lineages. It expands indications to autoimmune conditions involving T, NK, B and plasma cell lineages.

Core Advantages
  • Healthy donor-derived, overcoming autologous manufacturing limitations
  • CRISPR-Cas9 gene editing enhances potency and prolongs in vivo persistence
  • LNP-KO process avoids loss of T-cell function post electroporation
  • CRISPR-Cas9 gene editing enhances potency and prolongs in vivo persistence
  • Single-batch capacity for 500–1000 patients; cost equals 1/10 to 1/30 of autologous CAR-T
Pipeline Application
Skye-02

Skye-02 (CD19/BCMA dual-targeting, autoimmune diseases: Multiple Sclerosis (MS) and Rheumatoid arthritis (RA))

Fully-human Dual targeting 4th Generation QuickCAR Platform

QuickCAR Platform is a self-developed next-generation CAR-T manufacturing technology platform by Skye Medicine, designed to address the core bottleneck of long production cycles (7–14 days) for conventional autologous CAR-T cell products.

Through optimized cell activation and gene transduction workflows, this platform drastically shortens the CAR-T manufacturing cycle from the traditional 7–14 days to only 1–3 days, substantially reducing patients’ waiting time for treatment. Meanwhile, the QuickCAR Platform integrates the 4th-generation Armored CAR architecture. It enables intracellular autocrine expression of "enhancer" molecules, effectively supporting stemness maintenance and in vivo persistence of CAR-T cells, laying a technical foundation for deep and durable therapeutic responses.

Core Advantages
  • Fully human, dual-target, 4th-generation Armored CAR architecture that enhances cellular stemness and persistence
  • Proprietary T-cell metabolic reprogramming technology to mitigate in vivo CAR-T cell exhaustion and persistence limitations
  • Manufacturing turnaround of 1–3 days, significantly shortening patient waiting time
Pipeline Application
Skye-01

Skye-01 (CD19/CD20, B-NHL)

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