Much of the challenge of developing genetic medicines lies in having the right vector to deliver the therapy to the cells within the body where they need to go. 4D Molecular Therapeutics has developed platform technology that generates large numbers of genetically diverse, synthetic adeno-associated viral vectors that have desired characteristics using a process known as directed evolution. It is using these vectors to build a pipeline of genetic medicines across a broad set of conditions. We spoke to Alan Cohen, senior vice president of clinical development and therapeutic area head of pulmonology for 4DMT, about the limitations of existing vectors for genetic medicines, 4DMT’s directed evolution platform technology, and its programs in cystic fibrosis and Fabry disease.
Differentiating Gene Therapies through Regulatory Elements
How One Patient Organization Leverages Research Investments
Charging into the Storm
A Gene Therapy Developer that Embraces Different Models for Reaching Patients
Addressing a Shortage of Genetic Counselors with AI
A Small Molecule Therapy to Regenerate Muscle in People with DMD
Forging a Faster Path for Gene Therapies
Determining the Value of Rare Disease Therapies
Buying and Building a Gene Therapy Presence
A Clinical Trial Failure Derails a Promising Technology
Helping People with Undiagnosed Rare Diseases Find Answers
Speeding and Scaling the Development of Genome Editing Therapies
Getting a Next-Generation Genome Editing Therapy for Sickle Cell Disease Back on Track
Trying to Break a Leg in a Wheelchair
A Drug Developer that Makes Pediatric Cancers a Priority
Harnessing the Body’s Natural RNA Machinery to Treat Diseases
Targeting the Leading Cause of Death in Friedreich’s Ataxia with a Gene Therapy
How Inhaled mRNA May Help Rare Disease Patients Breathe Easier
Changing What’s Possible with Cell and Gene Therapies
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