Tessera Therapeutics Secures $41.3 Million ARPA-H Award to Develop Revolutionary In Vivo CAR-T Therapy
核心洞察
Tessera Therapeutics (搜索) received up to $41.3 million from ARPA-H (搜索) to develop single-dose in vivo CAR-T therapy (搜索) that engineers immune cells directly within the body.
The company's Gene Writing (搜索) platform aims to eliminate the need for ex vivo cell manipulation, viral vectors, and toxic lymphodepletion required by current CAR-T therapies.
Preclinical studies demonstrated robust T cell delivery and efficient CAR transgene integration using targeted lipid nanoparticles, supporting potential single-dose curative therapies.
Tessera Therapeutics (搜索) announced it has been awarded up to $41.3 million from the Advanced Research Projects Agency for Health (ARPA-H (搜索)) to advance its groundbreaking in vivo CAR-T therapy (搜索) platform. The funding, part of ARPA-H's Engineering of Immune Cells Inside the Body (EMBODY) program led by Program Manager Daria Fedyukina, Ph.D., will support development of a revolutionary approach that engineers functional CAR-T cells (搜索) directly within patients' bodies.
Revolutionary Gene Writing Technology
The biotechnology company is developing Gene Writers™, designed to precisely and durably integrate therapeutic instructions into the genome, enabling the incorporation of CAR sequences into T cells (搜索) using non-viral delivery methods. Through a single intravenous administration of Gene Writer formulated in a targeted lipid nanoparticle (搜索) (LNP), Tessera aims to generate potent, disease-specific CAR-T cells directly in the body.
This approach represents a significant departure from current CAR-T therapies, which require complex ex vivo cell manipulation, viral vectors, and toxic lymphodepletion procedures. "This work builds on the progress we've made using our Gene Writing (搜索) and delivery platforms to engineer T cells (搜索) in vivo and has the potential to dramatically improve the accessibility, safety, and scalability of currently available CAR-T therapies," said Michael Holmes, Ph.D., Chief Scientific Officer of Tessera Therapeutics (搜索).
Promising Preclinical Results
In preclinical studies, Tessera's targeted LNPs demonstrated robust delivery to T cells (搜索) and efficient CAR transgene integration, supporting the potential for single dose, curative therapies. The programs will leverage targeted LNPs designed to enable T cell specific delivery with high efficiency, addressing key limitations of current immunotherapy approaches.
Broad Therapeutic Applications
Under the EMBODY program, Tessera will develop in vivo CAR-T therapies for therapeutic applications in both oncology and autoimmune disease. The Gene Writing (搜索) platform is designed to write therapeutic messages into the genome by efficiently changing single or multiple DNA base pairs, precisely correcting insertions or deletions, or adding exon-length sequences and whole genes.
Platform Expansion
This award builds on Tessera's continued momentum across its pipeline, including recent advances in in vivo genome engineering for liver, hematopoietic stem cell, and T cell directed therapies. The company's proprietary lipid nanoparticle (搜索) delivery platform is designed to enable the in vivo delivery of RNA to targeted cell types, potentially transforming the treatment landscape for genetic diseases.
Founded in 2018 by Flagship Pioneering (搜索), Tessera Therapeutics (搜索) believes its Gene Writing (搜索) and delivery platforms will enable transformative genetic medicines to not only cure diseases that arise from errors in a single gene, but also modify inherited risk factors for common diseases and create engineered cells to treat cancer (搜索) and potentially autoimmune and other diseases.