Targeting Autoimmune Disease With Cell and Gene Therapy

Chair Introduction

Samik Basu, Chief Scientific Officer, Cabaletta Bio

3pm Presentation: Deconvoluting Cell and Gene Therapy For Autoimmune Disease

• The rationale for engineered T cell therapy as a potential curative approach for patients with treatment refractory disease
• Current state of the art and unique challenges and risks
• Near and mid-term developments to look out for

Samik Basu, Chief Scientific Officer, Cabaletta Bio

3.15pm Presentation: In vitro safety testing of MuSK-CAAR T cells

• Chimeric antigen receptor (CAR)-T cells are a promising therapy for the treatment of cancer and autoimmune diseases
• CAR-T cell-induced off-tumor/off-disease toxicities could potentially result in severe side effects, highlighting the importance of evaluating the safety of CAR-T cell therapies at an early stage of development
• In this case study, a human primary cell screen was performed to de-risk chimeric autoantibody receptor (CAAR) T cells targeting Muscle-specific tyrosine kinase (MuSK) autoantibodies-expressing B cells in patients with myasthenia gravis

Sabrina de Munnik, Senior Scientist, Charles River Laboratories

3.30pm Presentation: Pioneering RNA Cell Therapy for Autoimmune Diseases

• Descartes-08 is the first rCAR-T in clinical development for autoimmune disease. Descartes-08 is engineered by transfection of autologous CD8+ T cells with mRNA encoding anti-BCMA CAR.

• Conventional engineered CAR-T cell therapy uses DNA, which can be toxic. Effector function of cell therapy engineered with DNA amplifies exponentially with cell replication, frequently leading to uncontrollable PK/PD. Cartesian’s RNA approach designed to expand reach of cell therapy to autoimmunity with safer, potent, and less expensive therapies.

• In a phase 2a trial, Descartes-08 is observed to induce deep and durable clinical improvement in MG.

Metin Kurtoglu, Chief Operating Officer, Cartesian Therapeutics

3.45pm Presentation Title: KYV-101: a Fully Human Autologous CAR T-Cell Therapy for Autoimmune Diseases.

• KYV-101 is a fully human autologous anti-CD19 chimeric antigen receptor (CAR) T-cell therapy designed and demonstrated to have a favorable clinical safety profile
• The KYV-101 clinical development program will encompass clinical trials across multiple autoimmune diseases and is currently enrolling and planning trials in lupus nephritis, myasthenia gravis, and systemic sclerosis

James Chung, Chief Medical Officer, Kyverna Therapeutics