Therapeutic Target

 
News Articles for Therapeutic Target top ^
Dicerna Pharmaceuticals, Inc. (NASDAQ:DRNA), a leader in the development of RNAi therapeutics, today announced the presentation of new preclinical data on its Dicer substrate short interfering RNA (DsiRNA) therapeutics targeting MYC and β-catenin (CTNNB1) oncogenes in models of hepatocellular carcinoma (HCC) and colorectal cancer (CRC). The positive data, presented during a poster session on January 9 at MYC: From Biology to Therapy, a meeting of the American Association of Cancer Research (AACR) in La Jolla, Calif., provide additional insights into the parameters that impact the delivery and activity of DsiRNA therapeutics utilizing Dicerna’s proprietary EnCore™ lipid nanoparticle (LNP) technology in HCC and CRC tumor models.
Sign-up for Dicerna Pharmaceuticals Presents Positive Preclinical Data on its DsiRNA Therapeutics Targeting MYC and β-Catenin at AACR MYC: From Biology to Therapy Meeting investment picks
Alnylam Pharmaceuticals , Inc. (Nasdaq:ALNY), a leading RNAi therapeutics company, announced today that it has filed a Clinical Trial Application (CTA) with the U.K. Medicines and Healthcare products Regulatory Agency (MHRA) to initiate a Phase 1/2 clinical trial with ALN-CC5, a subcutaneously administered investigational RNAi therapeutic targeting complement component C5 for the treatment of complement-mediated diseases.
Sign-up for Alnylam Files Clinical Trial Application (CTA) for ALN-CC5, an RNAi Therapeutic Targeting Complement C5 in Development for the Treatment of Complement-Mediated Diseases investment picks
Alnylam Pharmaceuticals , Inc. (Nasdaq:ALNY), a leading RNAi therapeutics company, announced today that it has filed a Clinical Trial Application (CTA) with the Swedish Medical Products Agency (MPA) to initiate a Phase 1 clinical trial with ALN-AS1, a subcutaneously administered investigational RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS-1) for the treatment of hepatic porphyrias, including acute intermittent porphyria (AIP). Per the filed CTA, the Phase 1 trial of ALN-AS1 will be performed first in AIP patients who are asymptomatic “high excreters” (ASHE) – patients with a mutation in the porphobilinogen deaminase (PBGD) gene and elevated urinary aminolevulinic acid (ALA) and porphobilinogen (PBG) levels, but no recent symptoms of a porphyria attack – and then in AIP patients who experience recurrent porphyria attacks.
Sign-up for Alnylam Files Clinical Trial Application to Initiate a Phase 1 Study for ALN-AS1, a Subcutaneously Administered, Investigational RNAi Therapeutic Targeting Aminolevulinic Acid Synthase-1 (ALAS-1) for the Treatment of Hepatic Porphyrias investment picks
Alnylam Pharmaceuticals , Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today positive initial Phase 2 data with revusiran (ALN-TTRsc), an investigational RNAi therapeutic targeting transthyretin (TTR) for the treatment of TTR cardiac amyloidosis.
Sign-up for Alnylam Announces Positive Initial Phase 2 Data with Revusiran (ALN-TTRsc), an Investigational RNAi Therapeutic Targeting Transthyretin (TTR) for the Treatment of TTR Cardiac Amyloidosis, with up to 98.2% Knockdown of Serum TTR in Patients investment picks
Alnylam Pharmaceuticals , Inc. (Nasdaq:ALNY), a leading RNAi therapeutics company, and The Medicines Company (Nasdaq:MDCO), a global biopharmaceutical company focusing on saving lives, alleviating suffering and contributing to the economics of healthcare by focusing on the world’s leading acute/intensive care hospitals, announced today that Alnylam has filed a Clinical Trial Application (CTA) with the U.K. Medicines and Healthcare products Regulatory Agency (MHRA) to initiate a Phase 1 clinical trial with ALN-PCSsc, an investigational agent for the treatment of hypercholesterolemia.
Sign-up for Alnylam and The Medicines Company Announce Filing of a Clinical Trial Application to Initiate a Phase 1 Study for ALN-PCSsc, an Investigational, Subcutaneously Administered RNAi Therapeutic Targeting PCSK9 for the Treatment of Hypercholesterolemia investment picks
Alnylam Pharmaceuticals , Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, and The Medicines Company (Nasdaq: MDCO), a global biopharmaceutical company focused on hospital care, announced today that Alnylam has initiated a Phase 1 clinical trial with ALN-PCSsc, an investigational agent for the treatment of hypercholesterolemia.
Sign-up for Alnylam and The Medicines Company Announce Initiation of Phase 1 Clinical Trial for ALN-PCSsc, a Subcutaneously Administered Investigational RNAi Therapeutic Targeting PCSK9 for the Treatment of Hypercholesterolemia investment picks
Alnylam Pharmaceuticals , Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today that it has completed enrollment in its Phase 2 clinical trial with revusiran (re-VOO-si-ran), the recommended International Nonproprietary Name (INN) for ALN-TTRsc.
Sign-up for Alnylam Completes Phase 2 Enrollment and Initiates Open-Label Extension (OLE) Study with Revusiran (ALN-TTRsc), an Investigational RNAi Therapeutic Targeting Transthyretin (TTR) for the Treatment of TTR Cardiac Amyloidosis investment picks
Alnylam Pharmaceuticals , Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today it has initiated the ENDEAVOUR Phase 3 clinical trial of revusiran in transthyretin (TTR)-mediated familial amyloidotic cardiomyopathy (FAC). FAC is one of the predominant clinical manifestations of TTR-mediated amyloidosis (ATTR), and afflicts an estimated 40,000 people worldwide.
Sign-up for Alnylam Initiates ENDEAVOUR Phase 3 Clinical Trial with Revusiran (ALN-TTRsc), an Investigational RNAi Therapeutic Targeting Transthyretin (TTR) for the Treatment of TTR Cardiac Amyloidosis investment picks

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Therapeutic Target
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