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JPM 2025 Looks at the Cell and Gene Therapy Landscape – Autoimmune, Cancer, and Chronic Diseases are Front and Center

by Bernice Lottering
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The 2025 JPM Global Forum showcased cell and gene therapy advancements, highlighting FDA approvals, innovative treatments, and scalable production for wider accessibility.

At JPM 2025, companies like BlueRock Therapeutics, Ginkgo Bioworks, Tune Therapeutics, and Kyverna Therapeutics showcased their advancements in cell and gene therapies. BlueRock is progressing with stem cell therapy for Parkinson’s, while Ginkgo is enhancing cancer treatments through iPSC-derived therapies. Tune is focusing on chronic diseases, including Hepatitis B, and Kyverna is developing CAR-T therapies for autoimmune conditions like lupus. The GenScript-hosted forum highlighted these efforts and emphasized the critical role these therapies play in addressing major healthcare challenges.

BlueRock, Ginkgo Bioworks, and Tune Therapeutics Lead with 3 Groundbreaking Cell and Gene Therapies for Parkinson’s, Cancer, and Chronic Diseases

Topping the deals of the sector, BlueRock Therapeutics and Ginkgo Bioworks, alongside Tune Therapeutics, are advancing next-generation cell and gene therapies targeting major diseases. Here, BlueRock is advancing bemdaneprocel, an investigational pluripotent stem cell-derived therapy for Parkinson’s disease. Their Phase III exPDite-2 trial, set to begin in 2025, is the first registrational trial for an allogeneic stem cell therapy in Parkinson’s. This trial follows promising Phase I results and aims to assess efficacy, safety, and quality of life impacts in moderate Parkinson’s patients. The FDA’s RMAT designation for bemdaneprocel accelerates its clinical development.

Ginkgo Bioworks is collaborating with Universal Cells (an Astellas company) to optimize iPSC-derived cancer therapies. Ginkgo’s advanced screening and engineering platforms are designed to improve the persistence and potency of immune cell therapies for solid tumors. This collaboration aims to leverage Ginkgo’s high-throughput capabilities to accelerate the development of durable and scalable treatments, focusing on CAR-T cell therapies and other immune-engineered cells.

Meanwhile, Tune Therapeutics is advancing its epigenome editing platform, with a focus on chronic diseases. The company’s Tune-401, an epigenetic silencer for Hepatitis B, is currently in clinical trials in New Zealand and Hong Kong. Tune’s innovative approach to gene silencing holds promise for treating chronic conditions and reshaping the landscape of regenerative medicine. The latest $175M financing supports the growth of Tune’s pipeline, including its breakthrough approach to epigenetic therapies for long-term disease management.

Kyverna Charts Course for CAR-T Leadership in Autoimmune Diseases with KYV-101 at the Helm

Kyverna Therapeutics outlined its strategic priorities at JPM 2025, focusing on advancing CAR-T therapies for autoimmune diseases. CEO Warner Biddle emphasized stiff person syndrome (SPS) as the lead indication for KYV-101, aiming to make it the first CAR-T therapy approved for autoimmune conditions. Lupus nephritis and myasthenia gravis round out the company’s top targets, with SPS providing a rapid clinical entry point due to its unmet need and rare-disease status.

The company is advancing KYV-101 through pivotal trials, with SPS data expected in early 2026 and lupus nephritis results in late 2025. Kyverna’s pipeline also includes KYV-102, a next-generation candidate designed to simplify CAR-T manufacturing. The biotech is prioritizing capital efficiency and meaningful short-term milestones while considering strategic partnerships for future growth.

Interest in autoimmune CAR-T therapies is surging following breakthrough lupus studies. Competitors like like Bristol Myers Squibb’s CD19 NEX-T cell therapy asset for lupus nephritis, Novartis’ T-Charge platform for systemic lupus erythematosus, Cabaletta Bio’s autoimmunity-targeted T-cell engagers, and Cullinan Therapeutics’ proprietary CAR-T candidates are entering the field. 

Automation, Innovation, and Infrastructure for Smarter, Faster, and More Efficient Therapy Production

The 2025 JPM Global Forum, hosted by GenScript Biotech, spotlighted major advancements in cell and gene therapy. The FDA approved 42 new therapies, including Lantidra for Type 1 diabetes. Breakthrough islet replacement therapies are reshaping the medical landscape. With $10.9 billion invested in gene therapy in early 2024, the sector is experiencing rapid growth.

Moving forward, regulatory updates took center stage as the FDA outlined efforts to expedite gene therapy development. The agency is exploring faster approval pathways and harmonizing international regulations. Additionally, decentralized CAR-T manufacturing is expanding through automated systems. Advances in CRISPR technology and platform production methods are improving affordability and accessibility for rare disease treatments.

Finally, discussions focused on groundbreaking innovations redefining the future of cell and gene therapy. Dual CARs, TRUCK cells, and in vivo CAR-T therapies extend treatments to autoimmune diseases. Panelists emphasized automation and infrastructure as critical to scaling production. These innovations are crucial for improving efficiency and ensuring that life-saving therapies reach more patients.

JPM 2025 Outlined the Field of Cell and Gene Therapy with Breakthrough Treatments and Scalable Innovations

The 2025 JPM Global Forum highlighted significant advancements in the cell and gene therapy field. The conference spotlighted breakthrough therapies, including FDA-approved treatments like Lantidra for Type 1 diabetes. Key discussions focused on accelerating gene therapy development through faster approval pathways and international regulatory harmonization. Innovations such as dual CARs, TRUCK cells, and in vivo CAR-T therapies were showcased as cutting-edge solutions for both cancer and autoimmune diseases. Moreover, automation and infrastructure were emphasized as critical to scaling production, improving efficiency, and ensuring accessibility to life-saving therapies.

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