Member Companies Driving the Latest Advancements in Cell and Gene Therapy

It’s been a pivotal year for advancements in cell and gene therapy. Last December, the FDA approved the first medicine based on CRISPR technology, Casgevy, a groundbreaking treatment for sickle cell disease that was co-developed by Vertex Pharmaceuticals. In California, there has been recent buzz around multi-million dollar partnerships to increase production of CAR-T cell therapies, the debut of new facilities dedicated to cell and gene therapy research and breakthroughs that can help get treatments to patients with quicker turnaround times. Read on to find out more about the latest happenings.

A $380 Million Partnership for More Therapies

Cellares, a Bay Area company that’s known as a pioneer in automating the manufacturing process for cell therapies, hit a big milestone earlier this year and recently announced a major partnership. Its Cell Shuttle “factory in a box,” which the company says was designed to reduce labor and facility size requirements by 90 percent, received the current Good Manufacturing Practice (cGMP) approval in March. In April, Bristol Meyers Squibb signed a deal with Cellares for $380 million to increase production of its CAR-T cell therapies. Cellares said several Cell Shuttles at its facilities in the U.S., Japan and the European Union will be reserved exclusively for BMS. In California, the company currently has a 57,000-square-foot factory in South San Francisco.

Bayer Lauded for Groundbreaking Cell Therapy Facility in Berkeley

Bayer made headlines when it opened its $250 million Cell Therapy Launch Facility in Berkeley last October, as the 100,000-square-foot center is the company’s first manufacturing facility devoted to producing cell and gene therapies. The facility was recently named one of the top real estate development projects in the region by the San Francisco Business Times, and the company was also a finalist this month at the 2024 East Bay Innovation Awards for its collaborative community investments in Berkeley. On the clinical side, Bayer commenced Phase 2 of its clinical trial earlier this year for its investigational gene therapy, AB-1002, to treat congestive heart failure.

Sonoma Biotherapeutics Debuts in the Northwest

Sonoma Biotherapeutics, a Bay Area-based company which focuses on developing engineered regulatory T cell (Treg) therapies to for autoimmune disorders and inflammatory diseases, recently held a ribbon-cutting ceremony for its new R&D center in Seattle. The company took over four floors of a building on the Elliott Bay waterfront and the new lab space can accommodate 65 scientists. The new center has been in the works for two years and will focus on autoimmune research. Sonoma Biotherapeutics also recently launched a clinical trial to test an investigational cell therapy for rheumatoid arthritis.

Faster Therapies for Patients

Kite Pharma made a breakthrough in a new therapy for non-Hodgkin lymphoma when its CAR-T therapy, Yescarta, received FDA approval in 2017 to treat adults with certain forms of the disease. Yescarta works by using the patient’s own immune system to fight cancer. Earlier this year, the Los Angeles-based company received another FDA approval, this time for a new process to manufacture the therapy that cuts down production time from 16 days to 14 days. “For patients with relapsed or refractory large B-cell lymphoma, every day matters as the patient’s disease can be aggressive and worsen rapidly,” Cindy Perettie, executive vice president of Kite, said in a release. “Yescarta is the first and only treatment to demonstrate superior overall survival over the standard of care as a second-line treatment with curative intent for these patients, and today’s decision by the FDA allows us to further shorten our delivery time of Yescarta so that patients have the best possible chance of survival.”

Powering Quality Control for CRISPR

CRISPR QC, the San Diego-based company that developed the CRISPR Analytics Platform and CRISPR-Chip as quality control checks for gene editing processes, secured a Series A funding round earlier this year and is expanding with a satellite office in Phoenix, Arizona. CRISPR QC says its CRISPR-Chip technology allows for the direct, real-time measurement of CRISPR activity, and that there will be a growing need for this information. “As the FDA just approved the first CRISPR therapy, its proven clinical utility will power the field’s growth. However, the brute-force methods to develop this therapy are expensive and cost prohibitive, and safety concerns regarding these therapies linger on. CRISPR QC’s Analytics Platform is needed now more than ever,” Ross Bundy, co-founder of CRISPR QC, said in a release.The company is also showcasing its technology this week at ACA 2024 – The Summit of Angel Investing in Columbus, Ohio.

Collaboration to Battle a Rare Pediatric Disease

X-linked agammaglobulinemia (XLA) is a rare genetic disorder that primarily affects males and is often diagnosed during infancy and childhood. It affects the patient’s immune system and leads to repeated bacterial infections in the ears, lungs, sinuses and skin, which can potentially be life-threatening as the patient isn’t able to produce the necessary antibodies. Some sources report that XLA occurs at a rate of 1 in 190,000 live births. Seattle Children’s Research Institute, the research division of Seattle Children’s Hospital, recently teamed up with Genezen, an Indianapolis-based contract development and manufacturing organization, to advance the hospital’s XLA gene therapy program. According to a release, the goal of the program’s therapy for XLA is to genetically modify stem cells from bone marrow that will allow the patients to produce new B cells and antibody-producing B cells to restore antibody production and bolster the immune system.

In the News

ThinkCyte is a Bay Area-based company that specializes in building advanced scientific instruments to enable better research, diagnostics and drug development in the cell and gene therapy space. It’s known for VisionSort, which the company says is the world’s first AI-based, dual-mode fluorescence and morphometric cell sorting platform, that uses its proprietary “Ghost Cytometry” technology to sort and identify cells with complex phenotypes. The company published a paper in the March issue of Cell Reports Methods about a new method for high-throughput drug discovery using both CRISPR screening and its Ghost Cytometry. The paper was done in collaboration with research groups from the University of Toyko and the University of British Columbia.

Focusing on Single-Cell Genomics

Abiosciences specializes in single-cell genomic sequencing and analysis and bioinformatics to study diseases at the molecular level to gain insights into possible new therapies. The Bay Area-based company is a 10x Genomics Certified Lab and recently presented at the 10x Genomics Symposium in April. Chris Kang, Abiosciences’ head of U.S. operations, spoke on the topic of “Identifying a key transcriptional program in human aortic dissection through deep single-cell analysis.”

If you are a member working in the cell and gene therapy space, consider joining our Cell & Gene Therapy Committee. The next meeting is happening in-person in both South San Francisco and San Diego on May 30, and there is an option to attend virtually.