Bipartisan leaders, patient advocates, and industry experts gather at Takeda's Los Angeles facility to strengthen California's commitment to the rare disease community.
LOS ANGELES, CA – The California Rare Disease Caucus, chaired by Democratic Caucus Chair and Assemblymember Rick Chavez Zbur (D-Hollywood), held its Winter Roundtable Tuesday at Takeda's Los Angeles manufacturing facility, bringing together legislators, patient advocates, and biopharmaceutical leaders to discuss the urgent challenges facing Californians living with rare diseases and the policy solutions needed to expand access to life-changing treatments. The event highlighted California's leadership in life sciences and its role in driving discovery, development, and delivery of innovative therapies.
"This issue is deeply personal for me," said Assemblymember Rick Chavez Zbur, Chair of the California Rare Disease Caucus. "My sister Jackie battled ALS, and I witnessed firsthand how difficult it can be to get a diagnosis, find the right treatment, navigate insurance coverage and grapple with the deficiencies in the social safety net to obtain in-home care. That's the reality for so many of the 2.4 million Californians dealing with rare diseases—whether it's hemophilia, cystic fibrosis, muscular dystrophy, or one of thousands of other conditions."
The roundtable centered patient stories, the scientific advancements underway, and the remaining barriers in research, development, and coverage. Participants also shared recommendations for strengthening California's rare disease policy framework in the year ahead.
"Millions of Californians are battling rare diseases, and events like the Winter Roundtable help us better understand what patients are facing and how the state can better support them," said Senator Catherine S. Blakespear (D-Encinitas), Co-Vice Chair of the California Rare Disease Caucus. "Thankfully, we live in a state with a thriving life sciences industry that is working to develop innovative drugs and treatments. As policymakers, we must work to support the life sciences, the development of new treatments and patients who are coping with rare and difficult diseases."
"I am grateful to be a part of the California Rare Disease Caucus to raise awareness, support innovation and research, encourage advocacy and elevate the voices of Californians living with chronic conditions," said Assemblymember Diane Dixon (R-Newport Beach), Co-Vice Chair of the California Rare Disease Caucus. "I know we all want to do more than simply listen to the heartbreaking stories of the challenges faced by those living with a rare disease. We all need to advocate together and move the discussion forward because rare diseases don't care about political party affiliation. For those with firsthand experience, we have seen the suffering and toll it takes on both the patients and their families–that struggle is universal."
"Today's insights from patients, researchers, and industry leaders will translate into legislative action," said Assemblymember Jessica Calozan (D-Los Angeles), Member of the California Rare Disease Caucus. "If we want patients to have access to breakthrough therapies, we must continue building a strong research and innovation pipeline here in California."
"Rare diseases like hemophilia, muscular dystrophy, and ALS are devastating for patients and their loved ones," said Lynne Kinst, Executive Director of the Bleeding Disorders Foundation. "The conversations we had today will directly shape how we support individuals who have been waiting far too long for treatments."
Participants included patient advocates, leaders from California Life Sciences, and experts from organizations involved in developing and delivering treatments for rare diseases. Together, they shared perspectives on the scientific, regulatory, and access challenges that shape the patient experience.
The roundtable was hosted at Takeda's Los Angeles plasma fractionation facility, a long-standing part of California's life sciences ecosystem. The site, which is expanding its capacity and adding new jobs, plays a key role in producing therapies for conditions such as hemophilia, primary immune deficiency, and rare genetic respiratory diseases.
"California has the best life sciences ecosystem in the world. We have the researchers, the innovators, the companies, and the commitment to lead on rare disease policy," said Assemblymember Zbur. "Today is about listening—to patient voices, to the challenges faced in obtaining care and in developing and delivering treatments, so that we can turn that into action in Sacramento."
The California Rare Disease Caucus is a bipartisan legislative caucus established in 2017 to bring public and legislative awareness to rare disease issues, ensure patients have a voice in shaping policy, educate legislators and staff, and provide opportunities for engagement with the rare disease community. For more information, visit www.cararediseasecaucus.org
Assemblymember Rick Chavez Zbur serves as the Democratic Caucus Chair for the California State Assembly and represents the 51st Assembly District, which includes Universal City, Hollywood, Hancock Park, West Hollywood, Beverly Hills, Westwood, West Los Angeles, Santa Monica, and other portions of Los Angeles.
CONTACT: Vienna Montague, (916) 319-2051, Vienna.Montague@asm.ca.gov