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    Company profile for Cure Rare

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    About

    Cure Rare Disease™ is developing genetic medicines that are unique to the individuals they are meant to treat. Our mission is to offer effective, life-saving treatments developed through collaborations with world-renowned researchers and clinicians, and in partnership with our generous donors.The first-of-its-kind, CRISPR-based therapeutic aims to stop the progression of Duchenne muscular dystrophy in a single-patient dosing...
    Cure Rare Disease™ is developing genetic medicines that are unique to the individuals they are meant to treat. Our mission is to offer effective, life-saving treatments developed through collaborations with world-renowned researchers and clinicians, and in partnership with our generous donors.The first-of-its-kind, CRISPR-based therapeutic aims to stop the progression of Duchenne muscular dystrophy in a single-patient dosing.

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    Cure RareUnited_States_of_America Flag
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    U.S.A
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    Address
    1575 Tremont St., Unit 1107, Boston, Massachusetts 02120
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    Telephone
    833-863-6663
    Client Email
    Email
    enquiries@pharmacompass.com
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    https://www.cureraredisease.org/
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    NEWS #PharmaBuzz

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    CIRM Funds $7.4M for LGMD2I/R9 Therapy Development
    CIRM Funds $7.4M for LGMD2I/R9 Therapy Development

    15 Dec 2025

    // BUSINESSWIRE

    https://www.businesswire.com/news/home/20251215136129/en/Cure-Rare-Disease-Awarded-%247.4-Million-CIRM-Grant-to-Advance-Gene-Therapy-for-Limb-Girdle-Muscular-Dystrophy-Type-2iR9

    BUSINESSWIRE
    15 Dec 2025
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    Cure Rare Disease Gets FDA Orphan Status to Treat LGMD2I/R9 Gene
    Cure Rare Disease Gets FDA Orphan Status to Treat LGMD2I/R9 Gene

    24 Sep 2025

    // PHARMAWEB

    https://www.pharmiweb.com/press-release/2025-09-24/cure-rare-disease-secures-fda-orphan-drug-designation-for-investigational-gene-therapy-to-treat-limb

    PHARMAWEB
    24 Sep 2025
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    Cure Rare Disease Gets FDA Feedback on LGMD2I Gene Therapy
    Cure Rare Disease Gets FDA Feedback on LGMD2I Gene Therapy

    23 Jun 2025

    // BUSINESSWIRE

    https://www.businesswire.com/news/home/20250623545882/en/Cure-Rare-Disease-Receives-FDA-Feedback-on-Limb-Girdle-2iR9-Gene-Therapy-Program-Following-Successful-Pre-IND-Meeting

    BUSINESSWIRE
    23 Jun 2025
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    Cure Rare Disease Welcomes New Leaders to Advance Therapies
    Cure Rare Disease Welcomes New Leaders to Advance Therapies

    09 Jun 2025

    // BUSINESSWIRE

    https://www.businesswire.com/news/home/20250609715614/en/Cure-Rare-Disease-Welcomes-New-Scientific-and-Strategic-Leaders-to-Advance-Therapies-for-Ultra-Rare-Conditions

    BUSINESSWIRE
    09 Jun 2025
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    Cure Rare Disease's FDA Orphan Drug Designation for SCA3 therapy
    Cure Rare Disease's FDA Orphan Drug Designation for SCA3 therapy

    14 May 2025

    // BUSINESSWIRE

    https://www.businesswire.com/news/home/20250514432257/en/Cure-Rare-Disease-Receives-Orphan-Drug-Designation-from-FDA-for-Investigational-Therapy-Targeting-Spinocerebellar-Ataxia-Type-3-SCA3

    BUSINESSWIRE
    14 May 2025
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