At the American Association of Pharmaceutical Scientists (AAPS) | @aapscomms) National Biotechnology Conference, our CEO, Dr. Emil Kakkis, took center stage to continue emphasizing the critical role of biomarkers in driving therapeutic advancements for the #RareDisease community. It’s undeniable that biomarkers directly measure the underlying cause of disease. Pursuing accelerated approval using disease biomarkers is critical for tackling conditions that were once considered untreatable. Thank you, Karen Quadrini for moderating, the #AAPS community and all those who joined us in San Francisco. #Innovation #NBC2024 #AcceleratedApproval #Biomarkers
Ultragenyx
Biotechnology Research
Novato, California 79,297 followers
Rare mission. Fearless team. Incredible possibilities.
About us
Leading with purpose. Every day. “Ultragenyx was founded to advance innovative medicines for rare and ultrarare diseases that have never been treated before. We are delivering transformative therapies across multiple indications, and we have one of the most robust and diverse clinical pipelines in rare disease. Our focus is on doing the right things for patients both during development and commercialization to deliver on the promise of these therapies in a way that's meaningful for rare disease communities.” Emil D. Kakkis, M.D., Ph.D. Chief Executive Officer and President We have an inclusive culture of value and respect. Ultragenyx brings novel products to those living with rare and ultrarare diseases with a focus on debilitating genetic diseases. Founded in 2010, we have rapidly built a diverse portfolio of approved therapies and products aimed at diseases with a high unmet medical need for which many have no approved therapies. It takes courage, care, talent, and dedication to make a meaningful impact for those living with rare diseases, their families and the rare disease community. We put our people first, so they can take care of the rare disease community. We purposefully cultivate and nurture a dynamic, supportive work environment where we encourage every team member to share their new ideas and help us unlock more possibilities. Our team is made up of exceptional and diverse people with a strong passion and commitment to helping those living with rare diseases. By embracing generosity, curiosity, inclusion, and humility we are constantly learning together—fostering an environment that supports profound growth and fulfillment. If you want to have a meaningful impact, do the best work of your career while having fun and growing professionally and personally, come join our team! Visit our community guidelines at: https://ultragenyx.co/community
- Website
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http://www.ultragenyx.com
External link for Ultragenyx
- Industry
- Biotechnology Research
- Company size
- 1,001-5,000 employees
- Headquarters
- Novato, California
- Type
- Public Company
- Founded
- 2010
- Specialties
- rare disease and ultra-rare disease
Locations
Employees at Ultragenyx
Updates
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We are beyond proud to announce that our CEO, Emil Kakkis, has been honored as one of the Most Admired CEOs by the San Francisco Business Times. Emil's visionary leadership, dedication and relentless pursuit of innovation have been the driving force behind Ultragenyx. His leadership has not only shaped the future of our company but has also paved the way for groundbreaking advancements for the #RareDisease community, inspiring us all to #GoBeyondEveryDay. Congratulations, Emil, on this remarkable achievement! #Ultragenyx #Innovation
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Every year on May 15th, the #RareDisease community comes together to raise awareness and recognize the resilience of communities affected by all types of #Mucopolysaccharidosis (#MPS) disorders. This #MPSAwarenessDay, we are proud to support the International MPS Network and honored to share the stories of the MPS community, who strengthen, inspire, and drive us toward a future where rare diseases like #MPS are better understood and treated. Join us in raising awareness at: https://impsn.ca/mpsday/ #MPSDAY2024 #ITSABOUTTIME
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It is our honor to be recognized as a Corporate Impact Honoree from the Ritter Center for their annual Under the Stars event on May 16th. This recognition is a testament to the dedication of our team and the impactful work of our UltraGiving employee group, led by Lisa Helton, Vice President, Head of Program and Portfolio Management. We believe in #GoingBeyondEveryDay to give back to our communities and support the Ritter Center's mission to end homelessness in the Marin community. We look forward to celebrating this achievement and continuing our partnership with the Ritter Center to make a positive difference! #Ultragenyx #ESG #CorporateResponsibility
Ritter Center proudly recognizes Ultragenyx as our Corporate Impact Honoree for our 2024 Under The Stars event on May 16, 5:30pm, San Rafael. We recognize Ultragenyx for its exceptional employee volunteerism, financial contributions, and steadfast support of Ritter Center's mission to combat chronic homelessness. Ritter Center and Ultragenyx serve the needs of vulnerable communities, sharing a common thread as both patients of rare diseases and people experiencing homelessness can feel invisible. Also, A generous anonymous donor has promised to match ALL donations up to $25,000 now through the end of the event! If you can't attend Under the Stars, please consider making a gift and double your impact. Donate today 🎇 https://buff.ly/48YD2zG 🎇 #Marin #Ultragenyx #UndertheStars #Endhomelessness #DaretoDream #inspirehope #fundraiseforchange
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Our 3rd ESG report is out! We've made a lot of progress over the last year for the #RareDisease community, patients and our employees. See our highlights: https://lnkd.in/emKfNXBc #ESG #CorporateResponsibility
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We couldn’t imagine a better way to ring in Zach’s 15th birthday than with the passage of The Zachary Thomas Newborn Screening Act, HB 77, in Alabama by Governor Kay Ivey. Congratulations to the patient advocates whose leadership made this happen. Swapna Kakani, MPH, Brooke Thomas and the entire AL RARE team, your diligent and long effort to improve #NewbornScreening in Alabama has inspired us all. This year, due to the tireless and persistent effort of coalitions of people who live with and care about #RareDiseases, Tennessee, and now Alabama, have seen great success in achieving Recommended Uniform Screening Panel (RUSP) alignment, bringing the total number of states to 13. Allie Ladd, Terry Jo Bichell and Governor Bill Lee, thank you for your meaningful contributions in Tennessee and for signing this significant legislation into law. The fight for equitable newborn screening continues. Early detection of rare diseases leads to prompt treatment that can help prevent permanent disability, developmental delay, and death. It’s important for states to screen for all conditions on the RUSP. This enormous impact is what drives our State Government Affairs team to #GoBeyondEveryDay to support RUSP alignment efforts. #RareDisease #NBS #RUSP
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“The mood now not to pay for accelerated approval drugs is completely wrongheaded. Biomarkers that are evidence of underlying disease are superior to clinical endpoints; these biomarkers are not a compromise. If we design drugs to target the source of disease, we’ll move toward curing the disease rather than simply addressing symptoms. This is where we need to go.” - Dr. Emil Kakkis This week, our CEO Dr. Emil Kakkis joined a distinguished panel at the Health Technology Innovation Policy Conference 2024 for discussions about the impact of the Inflation Reduction Act on the industry and the FDA’s regulation of biologics. Dr. Peter Marks, the Director of the Center for Biologics Evaluation and Research of the FDA addressed challenges with respect to regulation of cell and gene therapies, and mentioned several things that the FDA hasn’t leveraged to the full extent yet: ➡️ Maximal use of the accelerated approval pathway ➡️ Global harmonization of regulatory requirements to allow commercialization in other countries sooner ➡️ Faster communication between FDA and sponsors (“warp speed for rare diseases”) to shorten length of development timelines Thank you to Stanford Byers Center for Biodesign for hosting an insightful event, and to Jeff Allen, Peter Kolchinsky, Ken Drazan and Dr. Peter Marks for contributing your expertise and insights. #RareDiseases #HealthcarePolicy #Innovation
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"When we honor our sorrows as much as our joys, our failures as much as our successes, we create a worldview in which every part of us belongs." - Dr. BJ Miller, MD A sincere thank you to Dr. BJ Miller, a palliative care physician who joined the Ultragenyx team this week for our latest employee UltraTalk. In his talk, Dr. Miller explored the concept of happiness as something that is rooted in accepting all parts of ourselves. This acceptance of life’s highs and lows creates internal peace and is the key to true resilience and happiness.
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Juliet's story is one of many who are fighting the devasting challenges of #OsteogenesisImperfecta (OI). While Juliet and her family have faced unimaginable challenges both emotionally and physically, they've never given up, and neither will we. On #WishboneDay, we shine a light on these #RareDisease warriors and their families. Read Juliet’s story to learn why we #GoBeyondEveryDay for those living with OI: https://lnkd.in/eDErZ8TW #OIAwarenessWeek
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Advocacy leaders Matthew Ellinwood, Chief Scientific Officer at the National MPS Society, and Mark Dant, Executive Director of The Ryan Foundation for Rare Disease Research, wrote an insightful article emphasizing the necessity of policy revisions for #AcceleratedApproval, underscoring the unique nature of #RareDiseases and why they require a distinct approval pathway rather than being lumped into standard processes. View the article: https://lnkd.in/gCwwwmqy
To save lives, the FDA must change how it evaluates rare disease treatments
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