Where Progress Lives: This Month in California Life Sciences September 2026

Each month, California Life Sciences (CLS) President and CEO Mike Guerra shares what’s shaping the life sciences industry, highlights the conversations that matter most to members, and offers a window into what the CLS team is working on.

Last month, I wrote about a remarkable run of FDA approvals and what that concentration of breakthroughs says about the ecosystem that produced them. September carried that momentum forward and showed just how far our industry’s reach extends. The month’s news ran the full length of the life sciences pipeline, from students at an Oakland STEM fair discovering what science can do, to children and families receiving the first treatments ever approved for their diseases. In between came new clinical data against some of the most feared diagnoses in medicine, major investments in California’s research infrastructure, new capital for the next generation of companies, and a community working to connect every stage of the journey from discovery to patients.

AB 1887 Signed Into Law

Governor Gavin Newsom signed AB 1887 into law in September, marking a critical step forward for California’s rare disease community. This legislation eliminates prior authorization and step therapy delays for FDA-approved rare disease treatments, ensuring patients can access the care their doctors prescribe without unnecessary insurance red tape.

Finding a cure for a rare disease is one of the hardest challenges in science. It can take over a decade and billions of dollars to bring a single treatment to patients. For every therapy that succeeds, researchers face years of failed trials and setbacks along the way. When that perseverance finally pays off, insurance shouldn’t be the obstacle standing between a patient and the treatment they’ve been waiting for.

We’re grateful to Assemblymember Rick Zbur, Chair of the CA Rare Disease Caucus, for his tireless leadership on this issue. His commitment to rare disease patients — rooted in his own family’s experience — never wavered, and California families are better off because of it.

From Earlier Answers to First-Ever Treatments

The measure of our industry’s work is what it means for patients and families, and September brought meaningful progress at every step, from earlier detection to first-ever treatments.

In San Diego, Circular Genomics announced a collaboration with Altoida that pairs a blood-based biomarker test with a 10-minute, self-administered digital assessment of cognitive function. Building on Circular Genomics’ research published in Nature Medicine, which found that a blood-based signature can predict progression to symptomatic Alzheimer’s years before clinical onset, the companies aim to move earlier detection out of specialty clinics and into routine primary care, with the first pilots expected later this year. For the millions of families who will face an Alzheimer’s diagnosis, earlier answers can mean more time to plan, act, and access treatment.

The month’s clearest breakthrough came from Carlsbad, where Ionis Pharmaceuticals had already won FDA approval on September 3 for ZANVASTRO™, the first disease-modifying treatment for Alexander disease. On September 22, Ionis and partner Otsuka announced that the Phase 3 FUSION study of ulefnersen met its primary endpoint, a combined measure of function and survival, in people living with FUS-ALS, a rare and rapidly progressive genetic form of ALS. FUSION is the first FUS-ALS clinical trial ever to meet its primary endpoint, and ulefnersen is the first treatment to target the disease’s underlying genetic cause and show the potential to change its course. FUS mutations account for roughly half of ALS cases in children and young people, many of whom face respiratory failure within one to two years of their first symptoms. Otsuka now plans to discuss potential expedited pathways to approval with the FDA.

Families living with rare diseases also saw a historic first. On September 17, Novato-based Ultragenyx received full FDA approval for FAYUVI™, the first-ever treatment for Sanfilippo syndrome Type A. This fatal disease steadily takes away a child’s ability to think, speak, and move, and median life expectancy is just 15 years. In clinical studies, children treated with the one-time gene therapy scored substantially higher on measures of cognition than a comparable group of untreated children. It is Ultragenyx’s second gene therapy approval, and it followed a development path that changed hands when an earlier developer ran into funding constraints despite positive clinical data. That this therapy reached patients at all is a reminder of why reliable pathways to capital matter so much for rare disease innovation.

Investing in California’s Next Fifty Years

The breakthroughs coming out of California depend on companies choosing to build their future here, and in September, both the companies that built this industry and the next generation behind them made that choice clear.

At the end of August, I joined Gilead Sciences in Foster City to celebrate the groundbreaking of its new Chess biologics facility alongside CEO Daniel O’Day, Executive Vice President of Pharmaceutical Development and Manufacturing Stacey Ma, and Senior Vice President of Manufacturing Operations Paul Foley. The facility is part of Gilead’s planned $32 billion investment in U.S. research, development, and manufacturing. Gilead has long been a leader in biotechnology, and their investment shows their continued commitment to California.

Less than two weeks later, Gilead opened a new 182,000-square-foot Research Center at its Foster City headquarters. The center brings together oncology, inflammation, and research data science teams and will support approximately 325 employees, including 260 laboratory scientists. It builds on a California presence that already includes more than 7,400 Gilead employees.

The next generation of California companies is drawing investment as well. This month, San Diego-based CLS member ADARx Pharmaceuticals raised more than $440 million in its initial public offering, the third-largest biotech IPO of the year. ADARx develops RNA-based medicines, and its most advanced program, onvuzosiran, is in Phase 3 testing for hereditary angioedema, a rare genetic condition that causes painful swelling attacks. The proceeds will help carry that program through Phase 3 and advance earlier-stage programs in complement-mediated diseases.

Fellow San Diego member Iambic Therapeutics also filed to go public. Co-founded in 2019 by Caltech professor Tom Miller and Fred Manby, Iambic uses AI to design new medicines and has built partnerships with companies including Takeda, AbbVie, and Bayer. Its lead program, a targeted therapy for HER2-driven cancers designed to reach the brain, could move into a registrational trial as early as next year. It is a clear example of how a California university lab can grow into a company advancing new medicines for patients.

Whether it comes from a company that helped build this industry or one just entering the public markets, investment of this scale is a long-term bet on California’s talent, institutions, and scientific community. Our work is to make sure California remains a place where those bets pay off, for the companies making them and for the patients counting on what comes next.

Connecting Every Stage of Our Ecosystem

An ecosystem as strong as ours depends on connecting people at every stage of the journey, and in September, CLS worked to do exactly that.

On September 10, our team joined Kits Cubed for its sixth annual STEM Fair in Oakland, where families enjoyed games, hands-on activities, and live music. At our booth, students explored the creativity behind scientific innovation through a magnetic slime experiment, and we were honored to help spark curiosity in tomorrow’s innovators.

For the founders already building, we introduced our Fall 2026 FAST California cohort, the latest group of early-stage entrepreneurs to join our no-cost, equity-free program, which surrounds founders with the mentorship and expertise to take their next step. This group of 11 innovative companies spans cell therapy, drug discovery, rare disease, medical devices, and neuroscience, reflecting the depth and diversity of innovation advancing California’s life sciences ecosystem.

On September 16 and 17, the Cell & Gene Therapy Symposium returned for its third year, this time as a two-day program presented in partnership with UC San Diego. Together with the California Institute for Regenerative Medicine (CIRM) and the Gene Therapy Initiative at UC San Diego, we brought more than 15 sessions to the Salk Institute for Biological Studies. Experts explored in vivo gene editing, the path from bench to bedside, commercialization and manufacturing, and how to partner with the patients at the center of this work. In the same month a gene therapy delivered the first treatment for Sanfilippo syndrome Type A, the timing could not have been more fitting.

At the executive level, senior life sciences leaders gathered in Torrey Pines on September 2 for our California Leadership Forum. The afternoon of candid, peer-to-peer conversation moved from the political landscape shaping this election cycle to what it takes to lead an organization through real transformation, at every stage from early growth to enterprise scale. We are grateful to every speaker who shared their expertise and to everyone who joined us.

Our own leadership grew this month as well. We welcomed Joy Russell as Chair and Sarah Huoh as Vice Chair of the CLS Board of Directors, along with four new board members: Chee Lum, Charles McWilliams, Anja Preissmann, and Andrew Ratz. I am grateful for their commitment to our community and look forward to the work ahead.

Looking Ahead

September showed the full reach of California’s life sciences ecosystem. In a single month, California took a critical step forward for its rare disease community, and companies here moved toward earlier answers for Alzheimer’s, delivered a breakthrough against a genetic form of ALS, and brought a first-ever treatment to families who had waited decades for one. Established leaders committed to building their next chapter in our state, and a new generation of companies secured the capital to carry promising science forward. Behind all of it is a community that connects the student discovering science for the first time to the founder, the scientist, and the executive carrying that work forward. Sustaining those connections, and the policy environment that supports them, is how California will continue to lead for the next 50 years.

I am grateful for the colleagues and partners who make all of this possible, and I look forward to sharing more next month.

Mike Guerra is President and CEO of California Life Sciences (CLS), California’s most influential life sciences trade association, representing more than 1,300 member companies. He chairs the Council of State Bioscience Associations and sits on multiple industry boards, working to advance California’s position as the world’s leading life sciences innovation ecosystem. Mike champions effective public policy at the national, state, and local levels, and is a steadfast advocate for the entrepreneurs and businesses driving life sciences forward in California.