Ultragenyx Pharmaceutical Inc., a biopharmaceutical company, focuses on the identification, acquisition, development, and commercialization of novel products for the treatment of rare and ultra-rare genetic diseases in the United States. Its biologic products include Crysvita (burosumab), an antibody targeting fibroblast growth factor 23 for the treatment of X-linked hypophosphatemia, as well as tumor-induced osteomalacia; Mepsevii, an enzyme replacement therapy for the treatment of children and adults with Mucopolysaccharidosis VII; and Dojolvi for treating long-chain fatty acid oxidation disorders. The company is also developing DTX401, an adeno-associated virus 8 (AAV8) gene therapy clinical candidate for the treatment of patients with glycogen storage disease type Ia; DTX301, an AAV8 gene therapy product candidate for the treatment of patients with ornithine transcarbamylase; UX143, a human monoclonal antibody for the treatment of osteogenesis imperfecta; GTX-102, an antisense oligonucleotide for the treatment of Angelman syndrome; UX701, for the treatment of Wilson disease; and UX053 for the treatment of glycogen storage disease type III. Ultragenyx Pharmaceutical Inc. has collaboration and license agreement with Kyowa Kirin Co., Ltd.; Saint Louis University; Baylor Research Institute; REGENXBIO Inc.; Bayer; GeneTx; Mereo; University of Pennsylvania; Arcturus Therapeutics Holdings Inc., Solid Biosciences Inc.; and Daiichi Sankyo Co., Ltd. Ultragenyx Pharmaceutical Inc. was incorporated in 2010 and is headquartered in Novato, California.
IPO Year: 2014
Exchange: NASDAQ
Website: ultragenyx.com
| Date | Price Target | Rating | Analyst |
|---|---|---|---|
| 6/6/2024 | $56.00 → $67.00 | Neutral → Buy | Goldman |
| 4/22/2024 | $77.00 | Outperform | RBC Capital Mkts |
| 12/8/2023 | $72.00 | Overweight | Wells Fargo |
| 6/14/2023 | $96.00 | Outperform | Credit Suisse |
| 6/6/2023 | $60.00 → $80.00 | In-line → Outperform | Evercore ISI |
| 4/26/2023 | $114.00 | Overweight | Cantor Fitzgerald |
| 1/18/2023 | $90.00 | Buy | Canaccord Genuity |
| 12/30/2022 | $82.00 | Buy | H.C. Wainwright |
| 11/3/2022 | $50.00 | Neutral → Outperform | Robert W. Baird |
| 10/13/2022 | $50.00 | Neutral → Buy | Guggenheim |
Third quarter 2024 total revenue grew 42% versus prior year to $139 million, including Crysvita® revenue of $98 million and Dojolvi® revenue of $21 million Reaffirmed 2024 expected total revenue guidance of $530 million to $550 million Breakthrough Designation granted for setrusumab (UX143) in osteogenesis imperfecta DTX401 Phase 3 follow-up data demonstrated higher and faster 62% mean reduction in cornstarch in crossover patients with glycogen storage disease type Ia (GSDIa) NOVATO, Calif., Nov. 05, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious r
NOVATO, Calif., Oct. 29, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultrarare genetic diseases, today announced that it will host a conference call at 5:00 p.m. ET on Tuesday, November 5, 2024, to discuss its financial results and corporate update for the quarter ending September 30, 2024. The live and replayed webcast of the call will be available through the company's website at https://ir.ultragenyx.com/events-presentations. The replay of the call will be available for three months. About Ultragenyx Pharmaceutical Inc.Ultragenyx is a biopharma
NOVATO, Calif., July 25, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultrarare genetic diseases, today announced that it will host a conference call at 5:00 p.m. ET on Thursday, August 1, 2024, to discuss its financial results and corporate update for the quarter ending June 30, 2024. The live and replayed webcast of the call will be available through the company's website at https://ir.ultragenyx.com/events-presentations. The replay of the call will be available for three months. About Ultragenyx Pharmaceutical Inc.Ultragenyx is a biopharmace
Treatment with DTX401 resulted in a statistically significant reduction in daily cornstarch intake at Week 48 (p<0.0001) with maintenance of glucose control Company will host investor call today at 5:00 p.m. ET NOVATO, Calif., May 30, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) today announced positive topline results from the Phase 3 GlucoGene study (NCT05139316) evaluating DTX401, an investigational gene therapy for the treatment of patients aged eight years and older with glycogen storage disease type Ia (GSDIa). The study achieved its primary endpoint, demonstrating that treatment with DTX401 resulted in a statistically significant and clinically meaningful
First quarter total revenue of $109 million, Crysvita® revenue of $83 million and Dojolvi® revenue of $16 million Reaffirmed 2024 expected total revenue guidance between $500 million to $530 million, Crysvita revenue of $375 million to $400 million, and Dojolvi revenue of $75 million to $80 million Presented positive interim Phase 1/2 data from GTX-102 Angelman syndrome study demonstrating that Expansion Cohorts showed rapid, clinically meaningful improvement across multiple domains consistent with or exceeding Dose-escalation Cohort data at Day 170 NOVATO, Calif., May 02, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the d
NOVATO, Calif., April 25, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultrarare genetic diseases, today announced that it will host a conference call at 5:00 p.m. ET on Thursday, May 2, 2024, to discuss its financial results and corporate update for the quarter ending March 31, 2024. The live and replayed webcast of the call will be available through the company's website at https://ir.ultragenyx.com/events-presentations. To participate in the live call, please register by clicking on the following link (registration link) and you will be provided
Expansion Cohorts showed rapid, clinically meaningful improvement across multiple domains; improvements consistent or exceeding Dose-escalation Cohorts data at Day 170 Additional long-term data in Dose-escalation Cohorts showed increasing and sustained clinical benefit through Day 758 Company will host investor call at 8:00 a.m. ET NOVATO, Calif., April 15, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) today announced new data from the Phase 1/2 study of GTX-102 for the treatment of Angelman syndrome. Patients in Expansion Cohorts A & B treated with a set dose and regimen of GTX-102 showed rapid and clinically meaningful improvement across multiple domains
Conference call now scheduled for Monday, April 15 at 8:00 a.m. ET Updated registration link below NOVATO, Calif., April 12, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) today announced that the conference call to discuss new clinical efficacy and safety data from the Phase 1/2 study of GTX-102 for the treatment of Angelman syndrome that will be presented at the American Academy of Neurology Meeting (AAN), will now be taking place on Monday, April 15, 2024 at 8 a.m. Eastern Time (ET). The slides accompanying the call will be shared on company's website at https://ir.ultragenyx.com/events-presentations before the call. Conference Call and Webcast Information T
NOVATO, Calif., April 12, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) today announced that preclinical and clinical data, including new clinical efficacy and safety data from the expansion cohorts in the Phase 1/2 study of GTX-102 for the treatment of Angelman syndrome, will be presented at the 76th Annual American Academy of Neurology Meeting (AAN) being held April 13-18 in Denver and virtually. GTX-102 is an investigational antisense oligonucleotide delivered via intrathecal administration and is designed to target and inhibit expression of UBE3A antisense transcript (UBE3A-AS). Abstracts are available to view online at www.aan.com/events/annual-meeting-abstrac
2023 Total Revenue of $434 million, Crysvita® revenue of $328 million and Dojolvi® revenue of $71 million 2024 Financial Guidance: Total Revenue between $500 million and $530 million, Crysvita revenue of $375 million to $400 million, and Dojolvi revenue of $75 million to $80 million Year-end 2023 cash balance of $777 million and 2024 guidance for Net Cash Used in Operations expected to be less than $400 million NOVATO, Calif., Feb. 15, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultrarare genetic diseases, today reported its financial results fo
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NOVATO, Calif., Nov. 20, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultrarare diseases, today reported the grant of 20,820 restricted stock units of the company's common stock to 13 newly hired non-executive officers of the company. The awards were approved by the compensation committee of the company's board of directors and granted under the Ultragenyx Employment Inducement Plan, with a grant date of November 16, 2024, as an inducement material to the new employees entering into employment with Ultragenyx in accordance with Nasdaq Listing Rule 5635(c)(4
Phase 1/2 data show improvements across all domains and confirm that Phase 3 Aspire study is amply powered to establish efficacy of GTX-102 Phase 3 program on track to begin enrollment by end-of-year NOVATO, Calif., Nov. 09, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) today announced Phase 1/2 data in support of the Phase 3 Aspire study for GTX-102, its investigational antisense oligonucleotide for Angelman syndrome, that will be presented at the 2024 Foundation for Angelman Syndrome Therapeutics (FAST) Global Science Summit in Orlando, Florida. "Cognition is the building block for the development and ascertainment of many new skills across a range of the
Third quarter 2024 total revenue grew 42% versus prior year to $139 million, including Crysvita® revenue of $98 million and Dojolvi® revenue of $21 million Reaffirmed 2024 expected total revenue guidance of $530 million to $550 million Breakthrough Designation granted for setrusumab (UX143) in osteogenesis imperfecta DTX401 Phase 3 follow-up data demonstrated higher and faster 62% mean reduction in cornstarch in crossover patients with glycogen storage disease type Ia (GSDIa) NOVATO, Calif., Nov. 05, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious r
NOVATO, Calif., Oct. 29, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultrarare genetic diseases, today announced that it will host a conference call at 5:00 p.m. ET on Tuesday, November 5, 2024, to discuss its financial results and corporate update for the quarter ending September 30, 2024. The live and replayed webcast of the call will be available through the company's website at https://ir.ultragenyx.com/events-presentations. The replay of the call will be available for three months. About Ultragenyx Pharmaceutical Inc.Ultragenyx is a biopharma
NOVATO, Calif., Oct. 18, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultrarare diseases, today reported the grant of 22,405 restricted stock units of the company's common stock to six newly hired non-executive officers of the company. The awards were approved by the compensation committee of the company's board of directors and granted under the Ultragenyx Employment Inducement Plan, with a grant date of October 16, 2024, as an inducement material to the new employees entering into employment with Ultragenyx in accordance with Nasdaq Listing Rule 5635(c)(4
NOVATO, Calif., Oct. 07, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced that it has received Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA) for setrusumab (UX143) as a treatment to reduce the risk of fracture associated with osteogenesis imperfecta (OI) Type I, III, or IV in patients 2 years of age and older. "FDA designation of setrusumab as a Breakthrough Therapy emphasizes the seriousness of osteogenesis imperfecta and the impact of this disease on people and their families affected by this disorder," said
NOVATO, Calif., Oct. 03, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) today reported that the Phase 1/2/3 Cyprus2+ study of its UX701 gene therapy has demonstrated meaningful clinical activity as well as improvements in copper metabolism in Stage 1. Multiple responders have completely tapered off of standard-of-care treatment with responses seen in all three dose cohorts. The company plans to enroll an additional cohort in Stage 1 at a moderately increased dose and with an optimized immunomodulation regimen to enhance the efficiency and efficacy of the gene therapy, with the objective of having the majority of patients come off of standard-of-care treatment before
NOVATO, Calif., Sept. 26, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) today announced that it will present seven abstracts related to its ongoing late-stage program evaluating setrusumab (UX143) and osteogenesis imperfecta (OI), including a late-breaker oral presentation of the 14-month data from the Phase 2/3 Orbit study, at the American Society for Bone and Mineral Research (ASBMR) 2024 Annual Meeting. The meeting is being held September 27-30, 2024, in Toronto, Canada. "Presentations at this year's ASBMR meeting add to our growing knowledge of the real-world impact and burden of OI and underscore the urgent need for an innovative therapy for these patients," s
NOVATO, Calif., Sept. 18, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultrarare diseases, today reported the grant of 62,555 restricted stock units of the company's common stock to 16 newly hired non-executive officers of the company. The awards were approved by the compensation committee of the company's board of directors and granted under the Ultragenyx Employment Inducement Plan, with a grant date of September 16, 2024, as an inducement material to the new employees entering into employment with Ultragenyx in accordance with Nasdaq Listing Rule 5635(c)
Morgan Stanley 22nd Annual Global Healthcare Conference on September 4 Cantor Global Healthcare Conference on September 17 Bank of America Global Healthcare Conference on September 18 NOVATO, Calif., Aug. 29, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultrarare genetic diseases, today announced participation in three upcoming investor conferences. Morgan Stanley 22nd Annual Global Healthcare Conference (New York, NY) Wednesday, September 4, 2024, Emil Kakkis, M.D., Ph.D., CEO and President, will participate in a fireside chat and host 1x1 m
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Goldman upgraded Ultragenyx Pharma from Neutral to Buy and set a new price target of $67.00 from $56.00 previously
RBC Capital Mkts initiated coverage of Ultragenyx Pharma with a rating of Outperform and set a new price target of $77.00
Wells Fargo initiated coverage of Ultragenyx Pharma with a rating of Overweight and set a new price target of $72.00
Credit Suisse resumed coverage of Ultragenyx Pharma with a rating of Outperform and set a new price target of $96.00
Evercore ISI upgraded Ultragenyx Pharma from In-line to Outperform and set a new price target of $80.00 from $60.00 previously
Cantor Fitzgerald initiated coverage of Ultragenyx Pharma with a rating of Overweight and set a new price target of $114.00
Canaccord Genuity resumed coverage of Ultragenyx Pharma with a rating of Buy and set a new price target of $90.00
H.C. Wainwright resumed coverage of Ultragenyx Pharma with a rating of Buy and set a new price target of $82.00
Robert W. Baird upgraded Ultragenyx Pharma from Neutral to Outperform and set a new price target of $50.00
Guggenheim upgraded Ultragenyx Pharma from Neutral to Buy and set a new price target of $50.00
Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) today announced that the company will share the latest clinical data, regulatory progress and program next steps for GTX-102, its investigational antisense oligonucleotide for Angelman syndrome, on Wednesday, July 24, at the 2024 Angelman Syndrome Foundation (ASF) Family Conference and Research Symposium in Sandusky, Ohio.
Canaccord Genuity analyst Whitney Ijem maintains Ultragenyx Pharmaceutical (NASDAQ:RARE) with a Buy and maintains $111 price target.
Ionis Pharmaceuticals Inc (NASDAQ:IONS) released results Monday from the completed multiple ascending dose (MAD) portion of the Phase 1/2 open-label study of ION582 in people with Angelman syndrome (AS). AS is a rare neurodevelopmental disorder caused by a loss of function in the maternal UBE3A gene. It affects an estimated 1 in 21,000 people worldwide. The data demonstrated consistent and encouraging clinical improvement on assessing all functional domains, including communication, cognition, and motor function measures. Also Read: Ionis Pharmaceuticals Reveals Late-Stage Donidalorsen Data, Analyst Says Results Show Competitive Hereditary Angioedema Profile. Overall, 97% of peop
14 analysts have shared their evaluations of Ultragenyx Pharmaceutical (NASDAQ:RARE) during the recent three months, expressing a mix of bullish and bearish perspectives. The following table summarizes their recent ratings, shedding light on the changing sentiments within the past 30 days and comparing them to the preceding months. Bullish Somewhat Bullish Indifferent Somewhat Bearish Bearish Total Ratings 6 5 3 0 0 Last 30D 0 1 0 0 0 1M Ago 0 0 1 0 0 2M Ago 4 3 1 0 0 3M Ago 2 1 1 0 0 Providing deeper insights, analysts have established 12-month price targets, indicating an average target of $86.79, along with a high estimate of $127.00 and a low estimate of $45.00. Witne
Cantor Fitzgerald analyst Kristen Kluska reiterates Ultragenyx Pharmaceutical (NASDAQ:RARE) with a Overweight and maintains $115 price target.
Wedbush analyst Laura Chico reiterates Ultragenyx Pharmaceutical (NASDAQ:RARE) with a Neutral and maintains $45 price target.
https://endpts.com/biogen-is-pursuing-more-dealmaking-this-year-with-plans-to-spend-up-to-8B/
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NOVATO, Calif., July 12, 2023 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultrarare diseases, today announced that it has appointed Howard Horn as executive vice president and chief financial officer (CFO) effective October 16, 2023. Mr. Horn will be responsible for leading the finance, accounting, corporate strategy and investor relations functions. "Howard is joining Ultragenyx and its leadership team during a pivotal period as our robust late-stage pipeline is maturing and we are preparing for the next phase of company growth," said Emil D. Kakkis, M.D.,
NOVATO, Calif., March 14, 2023 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) announced that Eric Crombez, M.D., has been promoted to chief medical officer and executive vice president effective May 1, 2023, succeeding Camille Bedrosian, M.D., who will remain with the company in a full-time strategic advisory role. Dr. Crombez currently serves as Ultragenyx's chief medical officer for gene therapy and inborn errors of metabolism. "Eric has extensive expertise in the development and execution of clinical development programs for rare genetic disorders and has been a driving force for our entire gene therapy pipeline," said Emil D. Kakkis, M.D., Ph.D., chief executive of
NOVATO, Calif., April 22, 2022 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases, today announced the appointment of Amrit Ray, M.D., M.B.A. to the company's Board of Directors. Dr. Ray will serve on the Board's Research and Development Committee. "Dr. Ray brings to our Board of Directors decades of global experience as a physician researcher, biopharmaceutical executive, and champion for patient access to innovative treatments," said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. "His expertise
Accomplished biotechnology executive brings over 40 years of commercial and leadership experience across multinational pharmaceutical and biotechnology companies to ShouTi ShouTi Inc., a clinical-stage biopharmaceutical company replacing biologics with world-class small molecule medicines using advanced computational and structure-based technology, today announced that Daniel Welch has been named Chairman of the company's board of directors. "It is a true privilege to welcome Dan to ShouTi as Chairman of our board," said Raymond Stevens, Ph.D., Chief Executive Officer, ShouTi. "As an industry veteran with an outstanding track record in guiding companies across all stages of development an
Dr. Suliman brings over 25 years of drug development, deal-making and company building expertise as ReCode continues advancing its pipeline of novel genetic medicines using its powerful selective organ targeting (SORT) LNP delivery platform ReCode Therapeutics (the "Company"), a biopharmaceutical company pioneering disease-modifying genetic medicines using its proprietary LNP delivery platform, today announced the appointment of Shehnaaz Suliman, M.D., MBA, M.Phil., as chief executive officer and as a member of the board of directors effective today. Former CEO, David Lockhart, Ph.D., will transition to the role of chief scientific officer and will remain president and a member of the boar
NOVATO, Calif., June 30, 2021 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultra-rare genetic diseases, today announced the appointment of Corsee Sanders, Ph.D., to the Board of Directors, effective June 29, 2021. Dr. Sanders, an experienced veteran of the biopharma industry, will serve as an independent director. "Dr. Sanders' established experience in global clinical development and especially with new technologies will be instrumental as we further advance our ongoing clinical trials and continue to expand our rare disease pipeline of novel therapeut