Ultragenyx says three patients in phase 1/2 gene therapy study had positive results [MarketWatch]
Ultragenyx Pharmaceutical Inc. (RARE)
Last ultragenyx pharmaceutical inc. earnings: 2/13 04:01 pm
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Source: MarketWatch
Ultragenyx says three patients in phase 1/2 gene therapy study had positive results Ultragenyx Pharmaceutical Inc. RARE, -0.71% shares rose 2.5% in premarket trade Wednesday after the company said that the first dose cohort of three patients in a phase 1/2 gene therapy study had "positive longer-term safety and efficacy" results. Moreover, after 24 weeks, the first patient dosed decided to discontinue all alternate medication three weeks ago and "continues to do well," Ultragenyx Chief Executive Emil Kakkis said. While the first patient's urea formation rate was increased "substantially" over 24 weeks, the second and third patients did not show a clinically meaningful change in rate of urea formation over 20 weeks and 12 weeks, Ultragenyx said. The gene therapy, DTX301, is being developed for ornithine transcarbamylase deficiency, a genetic disorder that can cause acute and chronic neurological deficits and other toxicities; an estimated 10,000 patients have OTC deficiency worldwid
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News
- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) is now covered by analysts at Royal Bank of Canada. They set an "outperform" rating and a $77.00 price target on the stock.MarketBeat
- Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4)GlobeNewswire
- Ultragenyx (RARE) Meets Efficacy Goal in Neuro Disease Study [Yahoo! Finance]Yahoo! Finance
- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) had its "neutral" rating re-affirmed by analysts at Wedbush. They now have a $48.00 price target on the stock.MarketBeat
- Ultragenyx Announces Positive Interim Phase 1/2 Data in Patients with Angelman Syndrome After Treatment with GTX-102 [Yahoo! Finance]Yahoo! Finance
RARE
Earnings
- 2/15/24 - Beat
RARE
Sec Filings
- 4/19/24 - Form 4
- 4/18/24 - Form 4
- 4/15/24 - Form 8-K
- RARE's page on the SEC website