Our AMR collection is live

Read our dedicated collection that showcases companies developing new therapies designed to tackle the serious global threat of antimicrobial resistance.

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  • Chameleon Biosciences is developing next-generation adeno-associated virus vectors that elicit minimal immune responses, allowing repeat dosing and resulting in superior efficacies. These EVADER vectors have universal application for existing adeno-associated virus vectors, and Chameleon is seeking partners to advance its lead program in severe hemophilia B and to programs that target retinal cells.

    • Chameleon Biosciences
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  • With its lead phase 3 candidate Nefecon in development for the treatment of primary immunoglobulin A nephropathy and a late-stage orphan focused NOX program, Calliditas Therapeutics is pioneering new rare-disease therapies.

    • Calliditas Therapeutics
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  • Global biotechnology company BioMarin focuses on developing and commercializing innovative therapies through a range of modalities. The company’s diverse pipeline, spanning exploratory to marketed programs, relies heavily on its genetic and genomic expertise, manufacturing capabilities, and long-term partnering models.

    • BioMarin Pharmaceutical Inc.
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  • Funding for early-stage projects and global collaborations are aiming to breathe life into the fragile ecosystem for novel antibiotics.

    • Raveena Bhambra
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  • In the past decade, deals involving rare diseases have increased in size and value, supported by the growing number of opportunities based on novel therapeutic platforms.

    • Raveena Bhambra
    • DealForma, LLC
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  • Seattle’s SEngine is using patient tumor samples to derive cancer organoids for accurate ex vivo drug testing, target identification and drug development.

    • SEngine Precision Medicine
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The search for rare disease therapies

The search for rare disease therapies

Complex, genetic and rare diseases continue to pose challenges globally, but new targeted approaches and personalized therapies are accelerating progress in the field. Here we present rare diseases therapies in development.
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