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Find Clinical Drug Pipeline Developments & Deals by Alnylam Pharmaceuticals

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            Lead Product(s): Lumasiran

            Therapeutic Area: Genetic Disease Product Name: ALN-GO1

            Highest Development Status: Phase III Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable September 30, 2020

            Details:

            ILLUMINATE-B is now the seventh Phase 3 study of an RNAi therapeutic that has yielded positive results, and the first-ever study evaluating the safety and efficacy of lumasiran in children under the age of six, including infants.

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            Lead Product(s): Vutrisiran

            Therapeutic Area: Genetic Disease Product Name: ALN-TTRSC02

            Highest Development Status: Phase III Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable September 03, 2020

            Details:

            The Company will discuss the potential for a biannual subcutaneous dosing regimen for vutrisiran, as well as discuss ongoing Phase 3 development for patisiran and vutrisiran across the APOLLO-B, HELIOS-A, and HELIOS-B studies.

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            Lead Product(s): Vutrisiran

            Therapeutic Area: Cardiology/Vascular Diseases Product Name: Undisclosed

            Highest Development Status: Phase III Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Blackstone Life Sciences

            Deal Size: $150.0 million Upfront Cash: Undisclosed

            Deal Type: Financing August 17, 2020

            Details:

            The investment includes up to $70 million to support the ongoing HELIOS-B Phase 3 study of vutrisiran in ATTR amyloidosis patients with cardiomyopathy, and up to $80 million to support Phase 2 & Phase 3 development of ALN-AGT, in development for the treatment of hypertension.

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            Lead Product(s): ALN-HSD

            Therapeutic Area: Hepatology (Liver, Pancreatic, Gall Bladder) Product Name: ALN-HSD

            Highest Development Status: IND Enabling Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable August 03, 2020

            Details:

            Company has submitted a clinical trial authorization application to The Medicines and Healthcare Products Regulatory Agency in the U.K to initiate a Phase 1 study of ALN-HSD, an investigational RNAi therapeutic targeting HSD17B13 for the treatment of NASH.

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            Lead Product(s): Lumasiran

            Therapeutic Area: Genetic Disease Product Name: ALN-GO1

            Highest Development Status: Phase III Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable July 13, 2020

            Details:

            The MHRA’s decision is based on the evaluation of the effects of lumasiran in PH1 patients and its safety profile, including data from the ILLUMINATE-A Phase 3 study.

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            Lead Product(s): Patisiran

            Therapeutic Area: Genetic Disease Product Name: Onpattro

            Highest Development Status: Approved Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Taiba Healthcare

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Agreement July 08, 2020

            Details:

            The distribution agreement is focused on both ONPATTRO® and GIVLAARI®, the first-ever commercialized RNAi therapeutics, as well as another late-stage therapy in development for Primary Hyperoxaluria Type 1.

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            Lead Product(s): Patisiran

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Approved Product Type: Cell and Gene therapy

            Recipient: American Porphyria Foundation

            Deal Size: $0.3 million Upfront Cash: Undisclosed

            Deal Type: Funding June 02, 2020

            Details:

            The global competitive grants program aims to inspire patient advocacy groups to develop solutions that address critical unmet needs among the hATTR amyloidosis, acute hepatic porphyria and primary hyperoxaluria type 1 (PH1) rare disease communities.

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            Lead Product(s): Lumasiran

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Phase III Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable June 01, 2020

            Details:

            Alnylam will discuss full results from the ILLUMINATE-A Phase 3 study of lumasiran in a webcast conference call. Lumasiran is an investigational RNAi therapeutic targeting hydroxyacid oxidase 1, the gene encoding glycolate oxidase, in development for the treatment of PH1.

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            Lead Product(s): Lumasiran

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Phase III Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable May 26, 2020

            Details:

            U.S. Food and Drug Administration (FDA) has accepted the Company’s New Drug Application (NDA) for lumasiran, an investigational RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1).

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            Lead Product(s): VIR-2703

            Therapeutic Area: Infections and Infectious Diseases Product Name: Undisclosed

            Highest Development Status: Preclinical Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable May 04, 2020

            Details:

            Vir and Alnylam plan to advance VIR-2703 as an inhalational formulation for the potential treatment and/or prevention of COVID-19.

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