Alnylam gene-silencing therapy to treat kidney disorder succeeds in late-stage study [Reuters (UK)]
Alnylam Pharmaceuticals, Inc. (ALNY)
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Source: Reuters
Alnylam gene-silencing therapy to treat kidney disorder succeeds in late-stage study - Reuters 3 Min Read (Reuters) - Alnylam Pharmaceuticals Inc’s gene-silencing therapy for a rare kidney disorder met the main goal of a late-stage study on Tuesday, bringing the company a step closer to marketing the first approved treatment for the condition. The study tested Alnylam’s experimental drug, lumasiran, against placebo in patients aged six and above with primary hyperoxyluria type 1 (PH1), a life-threatening condition that is estimated to affect one in 58,000 people globally. Alnylam plans to file for the drug’s approval in the United States and Europe early next year and hopes to launch the drug before the end of 2020. The company’s president, Barry Greene, estimates the market opportunity for PH1 treatments to be over $500 million. Lumasiran works using a mechanism called RNA interference (RNAi) to target and “silence” the genetic material involved in making excess amounts of a chemical
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ALNY
Earnings
- 10/31/24 - Beat
ALNY
Sec Filings
- 11/12/24 - Form SC
- 11/8/24 - Form SC
- 10/31/24 - Form 10-Q
- ALNY's page on the SEC website