250 episodes

RARECast is a Global Genes podcast hosted by award-winning journalist Daniel Levine. It focuses on the intersection of rare disease with business, science, and policy.

RARECast RARECast

    • Business

RARECast is a Global Genes podcast hosted by award-winning journalist Daniel Levine. It focuses on the intersection of rare disease with business, science, and policy.

    Protalix Looks Beyond Biobetters

    Protalix Looks Beyond Biobetters

    Protalix Biotherapeutics plant cell-based platform has been a point of differentiation for the company, a faster and more scalable way of producing protein therapeutics. The company first product, an enzyme replacement therapy for Gaucher disease, offered a proof of its approach. The company is pursuing therapies for Fabry disease and cystic fibrosis, as well as inflammatory diseases, but it is also in the process of rethinking its strategy of biobetters. We spoke to Dror Bashan, CEO of Protalix, about the company’s manufacturing platform, its therapeutic pipeline, and its desire to move toward addressing unmet medical needs.

    • 24 min
    Driving N-of-1 Therapies for Ultra-Rare Disease Patients

    Driving N-of-1 Therapies for Ultra-Rare Disease Patients

    Antisense pioneer and Ionis Pharmaceuticals Executive Chairman Stanley Crooke has launched a nonprofit to design and deliver custom RNA-targeted therapies free of charge for individual patients with ultra-rare diseases. The organization, n-Lorem Foundation, will leverage Ionis’ technology platform to speed the discovery and development of custom antisense oligonucleotide. We spoke to Crooke about the genesis of n-Lorem, how it will determine whether to work with an individual patient or researcher, and the challenges for regularizing the process of designing n-of-1 therapies for ultra-rare disease patients.

    • 28 min
    Cutting the Time and Cost of Drug Discovery with MicroOrgans

    Cutting the Time and Cost of Drug Discovery with MicroOrgans

    One of the reasons for the clinical failure of drugs is that animal models often fall short as predictors of how a drug will work in humans. StemoniX believes it can cut the time and cost of drug discovery with its microOrgans that are engineered from human induced pluripotent stem cells. These microOrgans can be used for high throughput human drug screening without the need for drugs to enter a human subject. We spoke to Ping Yeh, CEO of StemoniX, about the company’s technology platform, how it works, and its recent agreement with the AI company Atomwise to discover and develop small molecule candidates to treat the rare neurological condition Rett syndrome.

    • 28 min
    Acquisition Combines Two Pipelines for Rare Skin Diseases

    Acquisition Combines Two Pipelines for Rare Skin Diseases

    In December, Castle Creek Pharmaceutical Holdings completed its acquisition of Fibrocell. The deal brought together two companies developing therapies for rare skin disorders. We spoke to John Maslowski, CEO of the combined company, about its late-stage gene therapy in development for recessive dystrophic epidermolysis bullosa, how it differs from what is conventionally thought of as gene therapy, and what the acquisition does to advance the pipeline of the two companies.

    • 29 min
    Programming Cells to Produce Neuroprotective Factors to Treat ALS

    Programming Cells to Produce Neuroprotective Factors to Treat ALS

    ALS is a progressive neurodegenerative disease. As motor neurons die, people with the condition grow weaker and lose motor function. For most, death occurs within three to five years of diagnosis. Brainstorm Cell Therapeutics is conducting a phase 3 clinical trial of its autologous stem cell therapy that programs cells taken from patients to produce neuro-protective factors that promote the survival of neurons. We spoke to Ralph Kern, chief operating officer and chief medical officer of Brainstorm Cell Therapeutics, about ALS, the company’s late-stage experimental therapy, and how its approach may provide benefits for patients with other neurodegenerative disease.

    • 18 min
    Origin's Rapid Path to Seek Approval for Treatment of Ultra-Rare Metabolic Condition

    Origin's Rapid Path to Seek Approval for Treatment of Ultra-Rare Metabolic Condition

    Earlier this month, Origin Biosciences initiated a rolling submission with the U.S. Food and Drug Administration for approval to market an experimental therapy to treat the ultra-rare metabolic condition molybdenum cofactor deficiency (MoCD) Type A. The condition manifests itself shortly after birth and leads to difficulty feeding and intractable seizures. Children with the condition have a median survival of three years and those who live longer suffer irreversible damage to their central nervous system. The rolling submission comes about a year and a half after BridgeBio acquired the therapy from Alexion Pharmaceuticals and formed Origin Biosciences to develop it. We spoke to Neil Kirby, CEO of Origin, about MoCD, how its synthetic enzyme co-factor works to treat it, and the plans for Origin beyond the submission for approval.

    • 20 min

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