Company Overview and News

 
Ultragenyx Reports Top-line Data From Gene Therapy Candidate

2018-09-28 zacks
Ultragenyx Pharmaceutical Inc. (RARE - Free Report) announced positive top-line safety and efficacy data from the second dose cohort and positive longer-term data from the first dose cohort of the ongoing OTC phase I/II study of its adeno-associated virus (“AAV”) gene therapy candidate — DTX301.DTX301 is being evaluated forthe treatment of ornithine transcarbamylase (OTC) deficiency.
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Ultragenyx to Present at Jefferies Gene Therapy Summit

2018-09-26 globenewswire
NOVATO, Calif., Sept. 26, 2018 (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 Emil D. Kakkis, M.D., Ph.D., the company's Chief Executive Officer, will present at the Jefferies Gene Therapy Summit on Thursday, September 27, 2018 at 8:00am ET in New York.
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Arcturus Therapeutics to Present at October Investor Conferences

2018-09-25 globenewswire - 3
SAN DIEGO, Sept. 25, 2018 (GLOBE NEWSWIRE) -- Arcturus Therapeutics Ltd. (NASDAQ: ARCT), a leading RNA medicines company, today announced that Joseph Payne will present at four investor conferences in October: Microcap Conference, Ladenburg Thalmann 4th Annual Healthcare Conference, LEERINK Partners Roundtable Series – Rare Disease & Oncology, and Chardan 2nd Annual Genetic Medicine Conference. In addition, Arcturus will be present at the invitation-only JLABS CEO Summit where Melinda Richter, Global Head, Johnson & Johnson Innovation, JLABS, will host a fireside chat with Arcturus CEO Joseph Payne.
ARCT 4502 RARE

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AGTC Appoints William Aliski to its Board of Directors

2018-09-24 globenewswire
GAINESVILLE, Fla. and CAMBRIDGE, Mass., Sept. 24, 2018 (GLOBE NEWSWIRE) -- Applied Genetic Technologies Corporation (NASDAQ: AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare diseases, today announced it has appointed William Aliski to the company’s Board of Directors.
AGTC RARE

 
Arcturus Therapeutics Expands Management Team

2018-09-21 globenewswire
SAN DIEGO, Sept. 21, 2018 (GLOBE NEWSWIRE) -- Arcturus Therapeutics Ltd. (NASDAQ: ARCT), a leading RNA medicines company, today announced the appointment of Kevin T. Skol as Senior Vice President of Business Development & Alliance Management, and Suezanne Parker, Ph.D., as Vice President of Translational Biology. In parallel with these appointments, KC Kummerfeld CPA is promoted to Vice President of Finance and Corporate Controller reporting to Interim CFO Andy Sassine.
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Ultragenyx's Shares Surge More Than 80% in the Year So Far

2018-09-20 zacks
The share price of Ultragenyx Pharmaceutical Inc. (RARE - Free Report) has surged 85.2% year to date against the industry’s decline of 4.7%.
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RARE / Ultragenyx Pharmaceutical Inc. 8-K (Current Report)

2018-09-17 sec.gov
rare-8k_20180917.htm UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 _______________
RARE

 
Ultragenyx Expands Leadership Team and Appoints Dr. Wladimir Hogenhuis as Chief Operating Officer

2018-09-17 globenewswire
NOVATO, Calif., Sept. 17, 2018 (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 expanded its leadership team with the appointment of Wladimir (Vlad) Hogenhuis, M.D., as Chief Operating Officer, effective September 28, 2018. In this newly created role, Dr.
RARE

 
CODA Biotherapeutics Launches with $19 Million to Tackle Neuropathic Pain

2018-09-12 biospace
Coda Biotherapeutics launched in South San Francisco with a $19 million Series A financing. Lead investors were MPM Capital and Versant Ventures. They were joined by Astellas Venture Management and Novartis.
OREXQ OREX RARE

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Your Daily Pharma Scoop: Karyopharm Buoyed, MediNova Succeeds, Ardelyx's Publication

2018-08-31 seekingalpha - 7
Karyopharm Therapeutics (KPTI) reported that they plan to present, at the Society of Hematologic Oncology (SOHO) Annual Meeting in Houston, data from their Phase 2b STORM study evaluating selinexor in heavily pretreated patients with refractory multiple myeloma (NYSE:MM). The presentation is scheduled on September 12-15.
EVFM AMGN XENE KPTI RARE BHC ARDX RRMS SPHS AVDL NVS EOLS BHS

 
RARE / Ultragenyx Pharmaceutical Inc. null

2018-08-30 sec.gov
begin 644 filename1.pdf M)5!$1BTQ+C4-)>+CS],-"C$P(# @;V)J#3P\+TQI;F5A#0IE;F1S M=')E86T-96YD;V)J#7-T87)T>')[email protected]*, T*)25%3T8-"B @(" @(" @#0HR M,B P(&]B:@T\/"]&:6QT97(O1FQA=&5$96-O9&4O22 X-"],(#8X+TQE;F=T M:" V,B]3(#,X/CYS=')E86T-"FC>[email protected][email protected]
RARE

 
Ultragenyx to Present at Upcoming Investor Conferences

2018-08-30 globenewswire
NOVATO, Calif., Aug. 30, 2018 (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 Shalini Sharp, the Company's Chief Financial Officer, will present at the following upcoming investor conferences:
RARE

1
RARE / Ultragenyx Pharmaceutical Inc. 8-K (Current Report)

2018-08-29 sec.gov - 1
rare-8k_20180827.htm UNITED STATES
RARE

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Your Daily Pharma Scoop: ESPR Phase 3 Positive, FDA Rejects Volanesorsen, Ozempic Succeeds

2018-08-29 seekingalpha
Positive top-line results from the pivotal Phase 3 trial of Esperion’s (ESPR) bempedoic acid/ezetimibe combination pill study (1002-053) has been reported by the company. This trial was conducted in a 380 high-risk patient group with maximally tolerated statins. The trial evaluated the efficacy and safety of the bempedoic acid 180 mg/ezetimibe 10 mg combination pill compared to bempedoic acid, ezetimibe or placebo.
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Ultragenyx Announces FDA Accepts Proposal to Submit an NDA for UX007 for the Treatment of Long-Chain Fatty Acid Oxidation Disorders

2018-08-29 globenewswire
NOVATO, Calif., Aug. 29, 2018 (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 the U.S. Food and Drug Administration (FDA) has accepted Ultragenyx’s most recent proposal to submit a New Drug Application (NDA) for UX007 for the treatment of long-chain fatty acid oxidation disorders (LC-FAOD) based on existing data.
RARE

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Silicon Investor Message Boards

This table lists all message boards related to RARE / Ultragenyx Pharmaceutical Inc. on message board site Silicon Investor.

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CUSIP: 90400D108