Earlier this year the Innovative Genomics Institute and the life sciences tools conglomerate Danaher launched a collaborative center to develop genome-editing therapies for rare and other diseases. The Danaher-IGI Beacon for CRISPR Cures seeks to address hundreds of diseases with a unified research, development, and regulatory approach. Their goal is to create a new model for the development of genomic medicines. We spoke to Fyodor Urnov, IGI’s director of technology and translation and director of the new Beacon center, about the evolution of gene editing technology, the challenges of a platform approach, and how the organization plans to share what it learns.
Restoring Hearing through Gene Therapies
Creating a Sustainable Reimbursement Model for Ultra-Rare Therapies
Lumos Hopes Its Oral Alternative to HGH Will Fuel Its Growth
Advancing a Gene Therapy for a Rare and Fatal CNS Disorder
Addressing the Barriers to Patient Participation in Clinical Trials
A Venture Philanthropist Makes the Case for Advocates Taking Equity
Seeking a Sustainable Business Developing on N-of-1 Therapies
How One Drugmaker Reaches out to Communities at Greater Risk for Rare Heart Condition
A Next-Generation RNA Therapy Targets Telomere Disorders
BridgeBio Advances Therapy for Limb-Girdle Muscular Dystrophy that Started with Two Patient Families
Translating Urgency into Science
Empowering Ultra-Rare Disease Patients to Pursue the Discovery of Treatments
Why a Topical Cannabidiol Gel May Help Treat the Behavioral Symptoms of Fragile X
An Ultra-Rare Disease Drug Developer Tries to Navigate Regulatory Uncertainty
Powering Cells in People with Rare Mitochondrial Diseases
Powering Weakened and Stressed Cells in ALS to Function Better with Nanocrystal Therapy
Developing a New Class of Therapies Based on a Natural Cargo Carrier
Reaching Beyond the Limits of Enzyme Replacement Therapies with Gene Therapies
One Woman’s Journey as a Caregiver to a Husband with Frontotemporal Dementia
Treating Rare Endocrine Disorders with Therapeutic Peptides
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