Stylus Medicine Raises $85 Million! The In Vivo CAR-T Company Receives Investment from Eli Lilly and Johnson & Johnson

On May 12, Stylus Medicine announced it has raised $85 million (approximately 611 million RMB) to develop a technology aimed at tackling three "holy grail" level challenges in the fields of cell and gene therapy—targeted gene insertion, non-viral DNA delivery, and on-demand solutions for cell therapy.

Stylus Medicine, based in Cambridge, Massachusetts, was founded in 2022. It previously raised an undisclosed $40 million Series A round from RA Capital Management and Khosla Ventures. Recently, it secured an additional $45 million in funding, with investors including RA Capital, Eli Lilly, Johnson & Johnson, Chugai, Khosla Ventures, Myeloma Investment Fund, and Tachyon Ventures.

Stylus has developed a novel gene editing technology based on viral proteins (called recombinases). These proteins naturally insert large DNA fragments into bacterial genomes, but Stylus has used machine learning to design recombinases that can target specific regions of the human genome. This technology, based on research from scientists Patrick Hsu at the University of California, Berkeley, and the Arc Institute, is expected to be simpler and safer compared to CRISPR-based gene editing technologies. It only requires two components: mRNA encoding the recombinase and therapeutic DNA.

Stylus is encapsulating these two molecules in the same lipid nanoparticle to develop an easy-to-administer infusion therapy that can reprogram immune cells to target tumors. Currently, only a few biotech companies have initiated clinical trials for so-called in vivo cell therapy, but dozens of companies are racing to join the wave. Nevertheless, Stylus believes its technology will give it a competitive edge.

The company's initial focus is on developing in vivo CAR-T cell therapies. Stylus' approach enables precise and durable CAR-T cell generation within the body. These CAR-T treatments utilize lipid nanoparticles (LNPs) that target cells to directly deliver therapeutic payloads to immune cells within the body. This method has the potential to change patient treatments, increase accessibility, and redefine the possibilities of medicine.

Currently, therapies used by several companies require extracting cells from the patient, modifying them in the lab, and then re-infusing them into the patient after chemotherapy pre-treatment. This method has saved thousands of lives but is costly and complex. To break this barrier, Nuwaysir states that these therapies need to meet five criteria: It must be an in vivo cell therapy; it must be "as simple as possible," minimizing steps for modifying cells; it must be non-viral to reduce immunogenicity and production issues; it must be durable, capable of making permanent changes to immune cell genes; and the editing system must be able to deliver large gene payloads.

The company's large serine recombinase is key to achieving these goals. A few years ago, Hsu and collaborators from Berkeley and Stanford began systematically searching genome databases for as many large serine recombinases as possible. Stylus further advanced this work and used artificial intelligence to design new recombinases that can insert genes into a previously undisclosed region of human DNA, which the company believes is a safe location for therapeutic gene insertion.

Stylus has a small team of about 30 to 40 people and plans to grow at a moderate pace, initially focusing on the cancer field.

Emile Nuwaysir is the Chairman and CEO of Stylus Medicine. Previously, Emile served as the CEO and Chairman of BlueRock Therapeutics, an independently operated subsidiary of Bayer Pharmaceuticals. As the founding CEO, Emile helped establish a platform company at the intersection of cell and gene medicine, developing a series of innovative therapeutic programs and managing the company's acquisition and operations with Bayer. Emile also served as the CEO of Ensoma and as the former chair of the Alliance for Regenerative Medicine, an international advocacy organization with over 400 member companies dedicated to advancing cell and gene medicines.

Jason Fontenot is the Chief Scientific Officer at Stylus Medicine. Before joining Stylus, Jason was the Chief Scientific Officer (CSO) at Sangamo Therapeutics, Chief Scientific Officer at Immusoft, Head of Exploratory Research at Juno Therapeutics, and Head of Immunology Research at Biogen. Jason led and contributed to the successful advancement of numerous small molecule drugs, biologics, and cell and gene therapy drug development projects. These efforts led to multiple new drug clinical trial applications (INDs), including projects currently in clinical development and two FDA-approved therapies—Breyanzi, a CAR-T cell therapy targeting CD19 for treating B-cell lymphoma, and Zinbryta, a monoclonal antibody targeting CD25 for treating multiple sclerosis.

Dr. Patrick Hsu, co-founder of the Arc Institute and a professor of Bioengineering at the University of California, Berkeley, is one of the company's founders. Patrick Hsu was the first graduate student of CRISPR gene-editing pioneer, Professor Feng Zhang. Other scientific founders include Stanford University professors Ami S. Bhatt, Michael C. Bassik, and Lacramioara Bintu.

Conclusion

Stylus' launch comes at a time when many investors are pulling back from the gene-editing field, seemingly weary of an expanding toolkit of gene-editing methods, many of which claim to replace previous technologies.

When Tome launched at the end of 2023 with $213 million, it claimed its technology for dragging large DNA segments into specific parts of the genome was "the final chapter of genomic medicine." But just eight months later, Tome announced it was running out of funds and shut down last fall. Its rapid collapse has cast a shadow over the prospects of new gene-editing companies.

Nevertheless, Stylus' $85 million funding is the largest raised by a gene-editing startup since Tome, slightly surpassing the $82 million raised by Averna Therapeutics (formerly Exsilio Therapeutics) last June to advance its genomic drug development.