Emil Kakkis, Ultragenyx: The development of rare disease therapies for neurologic genetic diseases
2026 CCDS Scientific & Patient Symposium Keynote Talk.
Chief Executive Officer and President, Ultragenyx Pharmaceutical Inc.
Search every verified Emil Kakkis interview, podcast appearance, and on-the-record quote — each transcript cross-checked by AI and human review to confirm speaker identity. Emil Kakkis, founder, president, and CEO of Ultragenyx Pharmaceutical, has discussed the company's work developing treatments for rare and ultra-rare genetic disorders, including its progress toward profitability and its approach to drug development. In a January 2024 interview, Kakkis stated that the company expected 20% revenue growth in 2024 and profitability by 2026. He described the company's strategy as focusing on diseases with clear biology and using accelerated approval pathways to reduce development costs and bring treatments to market faster, noting that Ultragenyx had four approved products in its first ten years. In an August 2024 presentation, Kakkis discussed the company's clinical trial designs for rare diseases, including dose titration methods and the use of flexible endpoints. He also addressed challenges in the field, saying that regulatory requirements have become more demanding over time, making development "harder and not easier." Kakkis recounted past regulatory hurdles, including an FDA requirement for a long-term monkey study for a brain-targeted therapy, and described the company's decision to open trial sites outside the U.S. following a safety issue with GTX-102, citing a conservative FDA stance. He emphasized the importance of caregiver input in defining meaningful outcomes for conditions like Angelman syndrome.
2026 CCDS Scientific & Patient Symposium Keynote Talk.
Emil Kakkis, MD, PhD, founder and CEO of Ultragenyx shares his story and discusses development of therapeutics.
In this episode, WCG’s President of Patient Advocacy, Steve Smith, talks with Emil Kakkis, MD, PhD, Chief Executive Officer of Ultragenyx Pharmaceuticals and President of the non-profit EveryLife Foundation for Rare Diseases organization. Known for his work in developing an enzyme replacement therapy for the rare disorder MPS I and his connection to the Ryan Foundation, Dr. Kakkis shares with us his experiences in being involved in bringing drugs to market and forming relationships with patients along the way.
In this episode, we speak with Dr. Emil Kakkis, a physician and scientist who has spent more than 30 years helping to advance research, treatment and policy for rare disease patients. He is also the founder of both the EveryLife Foundation for Rare Diseases and Ultragenyx, a life sciences company dedicated to developing innovative treatments for rare and ultra-rare diseases. Topics discussed: advanced tech and the promise it holds, the key factors in advancing rare therapies, issues standing in the way of treatment access and more. Editor's Note: Chronic conditions and rare diseases don’t d…
Dr. Emil Kakkis, Ultragenyx founder & CEO, joins 'Closing Bell Overtime' to talk drug trials and development, its path to profitability and more.
Not to be missed! This is the keynote address for the 2023 Cure GM1 International Hybrid Community Meeting. Changing the future for GM1 and other ultra-rare diseases requires new rules. Emil D. Kakkis, M.D., Ph.D. Founder and CEO, Ultragenyx Support our work for continued progress and dissemination of information. Donate: curegm1.org/donate
Emil Kakkis, MD, PhD | Ultragenyx The Development of Rare Disease Therapeutics: Compassion and Transparency For more information, visit https://cureangelman.org This recording is sponsored by FAST Australia Recorded December 3, 2022 in Miami, Florida Disclaimer: The information contained in these slides is provided for informational purposes only and should not be construed as medical, legal, financial, or any other type of professional advice. The views and opinions expressed in this presentation and on the following slides are solely those of the presenter and not necessarily those o…
A short-talk by Emil Kakkis (Ultragenyx, USA) at Mitochondrial Medicine 2021. Organised by Wellcome Connecting Science Courses and Conferences. #mitochondria #mitochondrialdisease #mitochondria #genomics #scientificconference #keynotespeakers #keynotespeaker #keynotespeaking #eventswcs
Emil Kakkis, M.D., Ph.D., talks about the prospect of benefit and the challenges in drug development for rare diseases like Angelman syndrome. Disclaimer: The information contained in these slides is provided for informational purposes only and should not be construed as medical, legal, financial, or any other type of professional advice. The views and opinions expressed in this presentation and on the following slides are solely those of the presenter and not necessarily those of FAST. FAST does not guarantee the accuracy or reliability of the information provided herein. Under no circum…
Dr. Emil Kakkis, CEO and President and Founder of Ultragenyx Pharmaceutical Inc. joins Mary Woolley, President and CEO of Research!America, in a conversation about how we can get to answers faster for those living with rare diseases.
Emil Kakkis, MD, President and Founder of Ultragenyx Pharmaceutical Inc., discusses the company’s mission and how the company approaches treatment development for rare diseases like Mucopolysaccharidosis VII. The goal of Ultragenyx is to develop treatments for rare and ultra rare diseases. As Dr. Kakkis explains, one obstacle for the development of treatments for rare diseases is the small prevalence rates. He notes that having a small patient pool spread across the globe can cause said patients to feel isolated. For example, when Ultragenyx was developing vestroinidase alfa-vjbk (Mepsevvi) -…
Sign in to search the full transcript archive, filter by topic, and access every quote from Emil Kakkis.
The summary and quote tags on this profile are produced with AI assistance from verified, first-person interview transcripts, then checked by our team to confirm the speaker's identity and the accuracy of every quote. See how we verify →
Pro unlocks every transcript in full, plus unlimited search across every executive and company we cover.
50% off appliedPulling data at scale? The Data API gives you 30,000 transcripts a month over REST, MCP and JSON.
Compare all plans