Primary ciliary dyskinesia is a rare, genetic disease that arises from structural defects or the absence of the cilia lining of respiratory tract. This leads to mucus littered with trapped microbes, dust, and other debris getting caught in the airways, which can lead to permanent lung damage. Ethris is developing an inhaled mRNA therapy to get the body to produce a needed structural protein to restore normal cilia structure and function. We spoke to Thomas Langenickel, chief medical officer of Ethris, about how the company’s technology overcomes existing challenges for the therapeutic use of mRNA, its ability to deliver treatments directly to the lung, and its pipeline of therapies in development.
Using Plasma to Treat Rare Diseases
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
Using Directed Evolution to Develop New Vectors for Genetic Medicines
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
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