Date | Title | Description |
12.09.2024 | The Hidden Struggles of X-Linked Hypophosphatemia: A Call for Awareness and Action | X-linked hypophosphatemia (XLH) is a name that may not ring a bell for many. Yet, for those affected, it’s a daily battle. A recent analysis from the XLH Disease Monitoring Program (DMP) sheds light on the harsh realities faced by adults li... |
10.09.2024 | New Analysis Demonstrates Impaired Work Productivity and Increased Unemployment Rates in Adults with X-linked Hypophosphatemia (XLH) | Findings from XLH Disease Monitoring Program (DMP) published in Journal of Bone and Mineral Research (JBMR Plus)
DMP is a global effort, supported by Kyowa Kirin, to advance understanding and care of XLH through real-world research
Addition... |
09.09.2024 | More space for healthtech start-ups on the Novartis Campus |
Digitalisation is one of the megatrends in the healthcare sector. It not only promises greater efficiency, but also new possibilities in diagnostics and therapeutics. As this field requires expertise that traditional healthcare companies o... |
22.08.2024 | Global Genes' Annual Week in RARE Event Heading to Kansas City | Global Genes
Global Genes is taking Week In RARE on the road to Kansas City, after having hosted this annual event in California for over 12 years. Moving the location annually will help make the event more accessible and equitable to the g... |
05.06.2023 | Ultragenyx and Mereo BioPharma Announce Positive Data from the Ongoing Phase 2/3 Orbit Study of Setrusumab (UX143) in Osteogenesis Imperfecta (OI) | /EIN News/ -- Pediatric data show substantial induction of bone production in 1 week and a large increase in bone formation within 3 months of initiating monthly setrusumab treatment
Phase 3 sites beginning to screen patients
Conference cal... |
06.01.2023 | Ultragenyx Reports Preliminary 2022 Revenue; Guidance for 2023 Revenue and Cash Usage; Pipeline Updates and 2023 Milestones | Preliminary 2022 Total Product Revenue of $352 million to $356 million, Crysvita® Revenue in Ultragenyx Territories1 of $257 million to $258 million and Dojolvi® revenue of $55 million to $56 million
2023 expected Total Product Revenue guid... |
09.09.2022 | Ultragenyx and Mereo BioPharma to Present Setrusumab Data Update at ASBMR | NOVATO, Calif. and MOUNTAIN VIEW, Calif. and LONDON, Sept. 09, 2022 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), (“Ultragenyx”), a biopharmaceutical company focused on the development and commercialization of novel pro... |
11.02.2022 | Ultragenyx Pharmaceutical : Reports Fourth Quarter and Full Year 2021 Financial Results and Corporate Update - Form 8-K | Ultragenyx Reports Fourth Quarter and Full Year 2021 Financial Results and Corporate Update
2021 total revenue of $351.4 million and 2021 Crysvita® revenue in Ultragenyx territories1 of $192.6 million
2022 Crysvita revenue in Ultragenyx ter... |
11.02.2022 | Ultragenyx Reports Fourth Quarter and Full Year 2021 Financial Results and Corporate Update | 2021 total revenue of $351.4 million and 2021 Crysvita® revenue in Ultragenyx territories1 of $192.6 million
2022 Crysvita revenue in Ultragenyx territories guidance of $250 million to $260 million and Dojolvi revenue of $55 million to $65 ... |
05.01.2022 | GeneTx and Ultragenyx Provide Preliminary Update on Phase 1/2 Clinical Study of GTX-102 in Canada and U.K. Patients with Angelman Syndrome | SARASOTA, Fla. and NOVATO, Calif., Jan. 05, 2022 (GLOBE NEWSWIRE) -- GeneTx Biotherapeutics LLC and Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), companies partnered in the development of GTX-102, an investigational treatment for Angelman ... |
01.12.2021 | Ultragenyx Announces First Patient Dosed in Phase 1/2 Clinical Study of UX053, an mRNA Therapy for the Treatment of Glycogen Storage Disease Type III | NOVATO, Calif., Dec. 01, 2021 (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... |
02.11.2021 | Ultragenyx Reports Third Quarter 2021 Financial Results and Corporate Update | Third quarter 2021 total revenue of $81.6 million including Crysvita1 revenue to Ultragenyx of $50.3 million
2021 Crysvita revenue in Ultragenyx territories1 now expected towards the upper end of the$180 million to $190 million range previo... |
02.11.2021 | Ultragenyx Reports Third Quarter 2021 Financial Results and Corporate Update - Form 8-K | Ultragenyx Reports Third Quarter 2021 Financial Results and Corporate Update
Third quarter 2021 total revenue of $81.6 million including Crysvita1 revenue to Ultragenyx of $50.3 million
2021 Crysvita revenue in Ultragenyx territories1 now e... |
09.09.2021 | MEDIA ADVISORY: Ultragenyx Announces Mepsevii® (vestronidase alfa) Receives Reimbursement Approval for Treatment of Mucopolysaccharidosis VII in Italy | NOVATO, Calif., Sept. 09, 2021 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc., a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultra-rare genetic diseases, today annou... |
19.05.2021 | ULTRAGENYX PHARMACEUTICAL INC.
Ultragenyx Pharmaceutical : Corporate Presentation | Corporate Deck
May 19, 2021
Legal Warning
Cautionary note regarding forward-lookingstatements: This presentation contains forward-looking statements, including, but not limited to, statements regarding our expectations and projections regar... |
04.05.2021 | ULTRAGENYX PHARMACEUTICAL INC.
Ultragenyx Pharmaceutical : Strong Dojolvi launch continues with approximately 180 patients on reimbursed commercial therapy in the United States | Strong Dojolvi launch continues with approximately 180 patients on reimbursed commercial therapy
in the United States
NOVATO, Calif. - May 4, 2021 - Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the d... |
04.05.2021 | ULTRAGENYX PHARMACEUTICAL INC.
Ultragenyx Pharmaceutical : Reports First Quarter 2021 Financial Results and Corporate Update | First quarter 2021 total revenue of $99.4 million and Crysvita1 revenue to Ultragenyx of $42.1 million
2021 Crysvita revenue in Ultragenyx territories1 guidance of $180 million to $190 million reaffirmed
Strong Dojolvi launch continues with... |
08.02.2021 | Former Galena chief Mark Ahn back in hot water as DOJ levels insider trading charges for consultant work | When Mark Ahn departed troubled Galena Pharmaceuticals back in 2014, he told investors he looking to pursue other “long-held” goals. Turns out one of those goals may allegedly have been peddling stock based on insider info... |
02.07.2020 | FDA approvals roundup: Zogenix's Fintepla, Ultragenyx's Dojolvi, and Genentech's Phesgo | A weekly update on new drug approvals and indications from the FDA:
New approvals Fintepla gets the go-ahead for Dravet syndrome
Zogenix’s Fintepla (fenfluramine) has been approved for the treatment of seizures associated... |
15.05.2020 | Rare disease drugmaker Ultragenyx builds case for its gene therapy franchise with fresh trial updates | Focused on combating a smattering of rare diseases with an arsenal of biologics, small molecules, and gene therapies, Ultragenyx Pharmaceutical this week issued some updates on two of its experimental gene therap... |
04.05.2020 | New Darzalex formulation wins FDA approval; Israeli biotech plans $50M IPO | → During the Covid-19 crisis, drugs that must be administered in hospital settings or take hours to be infused are seeing delayed uptake as health care facilities are filled to the brim with coronavirus patients. Cancer d... |
14.10.2019 | FDA requests additional time for Flexion's sNDA; Genentech releases data for PhIII study of Rituxan | → In 2017, the FDA greenlighted Flexion Therapeutics‘ intra-articular therapy, Zilretta, designed as a treatment for osteoarthritis-related knee pain. Now, two years later, the company has announced that the FDA need... |
29.08.2018 | A former failure in PhII, the go-go FDA says it’s ready to consider an accelerated shortcut for Ultragenyx's UX007 | More than a year after Ultragenyx $RARE announced the failure of a Phase II study for its seizure medication UX007, the rare disease specialist says the FDA has now offered a thumbs-up for its willingness to consider an accel... |
18.09.2017 | Bidding war erupts over Dimension’s gene therapy pipeline as Ultragenyx counters Regenxbio deal | Emil Kakkis, Ultragenyx
A bidding war has suddenly erupted over one of the most damaged biotechs in gene therapy R&D.
Just weeks after Regenxbio lined up the acquisition of Dimension Therapeutics $DMTX for $86 million... |
19.04.2017 | Ultragenyx, Kyowa Hakko Kirin plan a date with the FDA after positive PhIII for X-linked hypophosphatemia | Emil Kakkis
Shares of Ultragenyx surged about 10% after the biotech reported clearly positive Phase III data for their Kyowa Hakko Kirin-partnered drug for X-linked hypophosphatemia. And now they plan to hustle that data pack... |
10.04.2017 | Are pharma industry companies converting patients with orphan diseases into lobbyists? | “Obviously, we wanted to send as many patients to advocate on Capitol Hill as possible,” said Yale, the director of the International Pemphigus & Pemphigoid Foundation.
Yale collected $7,500 in donations from a specialty pharmacy and a ... |
29.06.2016 | The biotech IPO feast flags as investors lose their appetite for R&D risk | There was a time after the 2008 crash that biotech IPOs were about as popular as snow in March. But from 2013 through 2015 generalist investors got over their fears and jumped into one of the hottest stock games on the planet. Fund... |
21.04.2015 | Dimension Therapeutics Nets $65M in Series B |
CAMBRIDGE, MA, Rare disease company advancing novel, liver-directed treatments for diverse genetic disorders, today announced the successful completion of a Series B financing.
>> Click here for more funding data on Dimension Thera... |
11.03.2015 | Diagnosis of rare genetic diseases receives a boost as Congenica gains £2m Genomics England SBRI award to fast-track technology for gene-disease interpretation | Diagnosis of rare genetic diseases receives a boost as Congenica gains £2m Genomics England SBRI award to fast-track technology for gene-disease interpretation |
23.06.2014 | Dimension Therapeutics Raises $30M Financing |
CAMBRIDGE, MA, Gene therapy company focused on developing novel treatments for rare diseases, today announced the closing of a $30 million financing.
>> Click here for more funding data on Dimension Therapeutics
>> To export... |
11.11.2013 | Rare-disease specialist Ultragenyx fires off a fast $86M IPO | Back in September, when Ultragenyx CEO Emil Kakkis was talking to FierceBiotech about the company for our annual Fierce 15 report, the biotech chief confidently predicted that he could take the company public next year. A few weeks later, t... |
14.08.2012 | Orphan drug firm with potential first treatment for HIBM raises $15 million | Solution/product: Ultragenyx is developing a sialic acid replacement therapy for treatment of the rare, progressive neuromuscular disease hereditary inclusion body myopathy. The disease is caused by a deficiency in sialic acid that causes m... |
20.06.2011 | Ultragenyx Pharmaceutical Closes $45M Series A Financing | Ultragenyx Pharmaceutical Inc., a Novato, CA-based developer of rare disease therapeutics, closed a $45m Series A financing.
The round was co-lead by TPG Biotech and Fidelity Biosciences, with participation from HealthCap and Pappas Venture... |
- | Ultragenyx Announces Results from Phase 1/2 Study of KRN23 in X-linked Hypophosphatemia in Adults | Source: Ultragenyx |
- | Ultragenyx Reports Second Quarter 2014 Financial Results and Corporate Update |
Second Quarter 2014 Financial Results
For the second quarter of 2014, Ultragenyx reported a net loss attributable to common stockholders of $13.6 million, or $0.45 per share, basic and diluted, compared with a net loss attributable to co... |
- | Are pharma industry companies converting patients with orphan diseases into lobbyists? | Wearing a blue T-shirt and worn jeans, Marc Yale stood near the welcome pole at the annual Rare Disease Legislative Advocates conference handing out money.
Envelopes in his left hand held $750 checks to help patients with a rare disease cov... |
- | Ultragenyx Announces Positive Results From a Long-Term Phase 1/2 Study of KRN23 in Adult Patients With X-Linked Hypophosphatemia | NOVATO, Calif., Sept. 15, 2014 (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 the presentation of r... |
- | Orphan drug firm with potential first treatment for HIBM raises $15 million | Company name: Ultragenyx Pharmaceutical Inc.
Industry: Pharmaceuticals.
Location: Novato, California.
Solution/product: Ultragenyx is developing a sialic acid replacement therapy for treatment of the rare, progressive neuromuscular disease ... |
- | Ultragenyx Announces Pricing of Public Offering of Common Stock | NOVATO, Calif., July 9, 2014 (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 the pricing of its unde... |
- | Ultragenyx Announces Initiation of a Phase 2 Study of KRN23 for Pediatric X-Linked Hypophosphatemia in the US and EU | Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, announced the first patient screened and enrolled in the Phase 2 study of the human mo... |
- | Ultragenyx Announces Initiation of Phase 3 Study of Recombinant Human Beta-Glucuronidase in Mucopolysaccharidosis Type 7 | Agreement Reached With Both FDA and EMA on Pivotal Trial Design
NOVATO, Calif., Dec. 15, 2014 (GLOBE NEWSWIRE) — Ultragenyx Pharmaceutical Inc. (Nasdaq:RARE), a biopharmaceutical company focused on the development of novel products for rar... |