12 Leading Experts Advancing Cystic Fibrosis Gene Therapy
When families receive a cystic fibrosis diagnosis—particularly those with CFTR mutations unresponsive to modulator drugs—finding the right gene therapy experts can mean the difference between disease progression and functional restoration. The field has advanced from laboratory concepts to clinical trials testing inhalable gene therapies in patients. This directory connects families to the physicians, researchers, and institutions making cystic fibrosis gene therapy accessible—and shows how Nome's platform delivers AI-generated, expert-reviewed reports that map real treatment options for rare CFTR variants in 30 days instead of months.
From UCLA's inhalable CRISPR nanoparticles to Oxford's inhaled gene therapy trials enrolling 40 to 120 participants, the experts profiled here are advancing gene therapy from preclinical validation to patient treatment. Nome partners with these leading researchers and coordinates the complex development pieces—geneticists, contract manufacturers, regulators—that families cannot navigate alone.
1. Dr. Donald Kohn, MD – UCLA
Institution: Department of Microbiology, Immunology and Molecular Genetics, UCLA
Research Focus: Inhalable gene therapy for cystic fibrosis using lipid nanoparticles
Dr. Kohn's team at UCLA is developing breakthrough CRISPR-based gene editing delivered via inhalable nanoparticles to correct CFTR mutations directly in lung cells. His work addresses what he describes as trying to "get into Fort Knox"—overcoming thick mucus, inflammation, and hard-to-reach cells at the bottom of airways.
Key contributions:
Leading preclinical studies demonstrating over 60% correction of target CFTR mutations in animal lung tissue models
Developing lipid nanoparticle delivery systems that shield gene-editing machinery from immune defenses
Collaborative approach integrating pediatric pulmonology, gene editing expertise, and nanotechnology
How Nome helps: While Dr. Kohn's academic lab focuses on preclinical validation and early-stage trials, Nome provides families with systematic access to emerging therapies through its secure platform that presents options with evidence and feasibility—no matter how rare the CFTR mutation. When families share genetic test results, Nome's AI analyzes dozens of research programs like UCLA's in minutes, mapping patient-specific eligibility.
2. Dr. Brigitte Gomperts, MD – UCLA
Position: Professor of Pediatrics and Pulmonary Medicine, UCLA
Lab Focus: Pediatric precision medicine and stem cell research
Dr. Gomperts brings critical pediatric pulmonology expertise to UCLA's gene therapy consortium. She emphasizes the collaborative puzzle-solving required: "You have to know" which cells you're targeting, how to reach them, and the best way to fix the faulty gene—and her team assembled all three pieces.
Active programs:
Identifying optimal target cell populations in pediatric CF lungs
Developing age-appropriate dosing protocols for inhaled gene therapies
Translating preclinical findings into pediatric clinical trial designs
For families: UCLA accepts consultations through pediatric pulmonology referrals. The team actively collaborates with patient advocacy organizations to identify trial candidates.
How Nome helps: Diagnosis expertise exists at major academic centers like UCLA, but development coordination does not. Nome's research partnerships accelerate the translation from bench science to bedside by coordinating the operational complexity—contract manufacturers, safety testing, regulatory submissions—that academic labs struggle to manage at scale.
3. Dr. Steven Jonas, MD – UCLA
Position: Assistant Professor of Pediatrics, UCLA
Specialty: Nanotechnology for gene therapy delivery
Dr. Jonas engineered the lipid nanoparticles enabling UCLA's inhalable gene therapy approach. His innovation: "Our nanoparticles" are the packaging that help transport the gene-editing machinery while shielding it from the body's defenses.
Technology achievements:
Developed nanoparticles that penetrate thick CF mucus and reach airway epithelial cells
Achieved efficient cellular uptake without triggering inflammatory responses
Demonstrated stable delivery of CRISPR-Cas9 components in preclinical models
How Nome helps: Advanced delivery technologies like Dr. Jonas's nanoparticles represent one therapeutic modality among many. Nome's platform evaluates whether lipid nanoparticle delivery, viral vectors (AAV), or mRNA approaches best suit each patient's specific CFTR mutation—then connects families to appropriate research teams with transparent timelines and feasibility assessments.
4. Dr. Eric Alton, MD, PhD – Imperial College London
Position: Professor of Gene Therapy and Respiratory Medicine, Imperial College London
Leadership: Principal investigator for UK CF Gene Therapy Consortium
Website: Oxford Medicine
Dr. Alton leads inhaled gene therapy clinical trials across the UK and Europe. His work aims to deliver "long-lasting CFTR function improvement and disease modification for people with CF irrespective of their mutation type."
Clinical trial details:
Phase I/II trials evaluating safety and efficacy over 24 weeks
Nebulized delivery targeting lung epithelial cells
Enrolling patients with CFTR mutations not responsive to current modulators
Mostly mild side effects including cough and transient irritation; serious adverse events rare
Recognition: Results presented at American Thoracic Society International Conference 2025, generating significant clinical interest.
How Nome helps: Clinical trials like Dr. Alton's often have narrow eligibility criteria and limited geographic access. Nome's AI-powered system analyzes patient genetic data against active trials worldwide, identifying matches within minutes and providing families with enrollment pathways—including trials in the UK, Europe, and US that manual literature searches would miss.
5. Dr. Patrick Mogayzel, MD, PhD – Johns Hopkins Medicine
Position: Director, Cystic Fibrosis Center, Johns Hopkins Medicine
Institution: Johns Hopkins Hospital
Dr. Mogayzel stated clearly: "In order to" truly cure cystic fibrosis, doctors must turn to genetic therapy. His center actively participates in gene therapy trials and provides comprehensive genetic counseling for CF families.
Johns Hopkins CF Center offerings:
Multidisciplinary care teams including pulmonologists, genetic counselors, and nutritionists
Access to emerging clinical trials through institutional partnerships
Patient education programs explaining gene therapy options
Shared decision-making framework for experimental therapy enrollment
Geographic reach: Mid-Atlantic families benefit from proximity; Hopkins accepts referrals nationally for complex cases.
How Nome helps: Major CF centers like Johns Hopkins provide excellent clinical care and trial access—but only for patients who can travel. Nome's provider network delivers genetic counseling and therapeutic assessments remotely, enabling families anywhere to receive expert-reviewed options without relocating. Nome then coordinates with institutions like Hopkins for execution when trials require onsite participation.
6. Cystic Fibrosis Foundation
Organization: Leading patient advocacy and research funder
Leadership: Michael P. Boyle, President & CEO
Website: cff.org
The CF Foundation committed $24 million to Prime Medicine in 2025 to advance gene editing therapies for cystic fibrosis—demonstrating venture philanthropy at scale.
Major funding initiatives:
Portfolio of gene therapy research grants across academic and commercial partners
Investment in manufacturing capacity for personalized therapies
Support for clinical trial infrastructure
Patient registry providing natural history data for endpoint validation
Impact: Foundation-funded research contributed to development of CFTR modulator drugs now serving most CF patients; gene therapy investment targets the 10-20% with null mutations who lack options.
How Nome helps: Patient advocacy organizations like the CF Foundation fund discovery and early development. Nome operationalizes that science for individual families—taking foundation-funded research breakthroughs and converting them into personalized treatment plans with transparent pricing and execution timelines. Families benefit from collective investment through individualized access.
7. Emily's Entourage
Organization: Patient-founded nonprofit funding CF research
Focus: Treatments for nonsense mutations (10-15% of CF patients)
Model: Family-founded venture philanthropy
Emily's Entourage funds research specifically targeting CF patients with nonsense mutations who cannot benefit from current CFTR modulators. The organization exemplifies the family-founded nonprofit model advancing gene therapy when traditional pharma investment remains unviable.
Research priorities:
Gene therapy approaches correcting nonsense mutations
Read-through drug development
Clinical trial readiness programs
Patient identification and registry development
How Nome helps: Organizations like Emily's Entourage represent the patient-founded determination Nome's founder Stevie Ringel shares—both told "there's nothing to do" and refused to accept it. Nome built technology to scale this determination: AI that analyzes genetic mutations in minutes instead of months, connecting families with nonsense mutations to relevant trials and custom therapy development pathways the moment diagnosis occurs.
8. 4D Molecular Therapeutics
Company: Clinical-stage gene therapy biotech
Platform: Targeted adeno-associated virus (AAV) vectors
CF Program: AAV-delivered CFTR gene replacement
4D Molecular Therapeutics develops AAV6.2-based gene therapies designed to transduce lung epithelial cells efficiently. Their CF program aims to deliver functional CFTR genes via nebulized administration, potentially offering single-dose treatment.
Development status:
Preclinical validation in CF animal models
Manufacturing partnerships for GMP AAV production
Regulatory pathway discussions with FDA
CF Foundation investment supporting clinical advancement
Platform advantage: Engineered AAV capsids optimized for lung tropism and reduced immunogenicity compared to natural serotypes.
How Nome helps: Companies like 4D Molecular represent one therapeutic approach among many. Nome's Operating System knows which contract manufacturers can produce AAV vectors, their lead times, and how to structure agreements—solving the operational complexity families cannot navigate manually. When AAV therapy fits a patient's mutation profile, Nome coordinates manufacturing, safety testing, and regulatory approvals across vendors.
9. Leading CF Gene Therapy Contract Manufacturers
Key Organizations:
Vigene Biosciences: AAV production platforms with CF-specific manufacturing experience
Catalent Pharma Solutions: GMP manufacturing suites supporting gene therapy scale-up
Oxford Biomedica: European AAV contract manufacturing with regulatory track record
Manufacturing requirements for CF gene therapies:
GMP-compliant production facilities
Vector potency assays validated for lung delivery
Quality control testing for genomic contaminants
Fill-finish operations compatible with nebulizer formulations
Cold-chain logistics for gene therapy stability
Bottleneck reality: Manufacturing capacity represents a critical constraint. Lead times for AAV production can extend 12-18 months; lipid nanoparticle platforms offer faster turnaround but require different quality specifications.
How Nome helps: This manufacturing complexity is precisely the bottleneck Nome solves. While academic labs spend months identifying appropriate manufacturers and negotiating contracts, Nome's AI knows which of 50+ contract manufacturers can produce specific CF gene therapies, their current lead times, and how to structure agreements—automating coordination that manual workflows cannot scale. Families get execution-ready manufacturing plans in weeks, not quarters.
10. FDA Office of Tissues and Advanced Therapies (OTAT)
Division: Center for Biologics Evaluation and Research (CBER)
Leadership: Dr. Peter Marks, Director, CBER
Focus: Gene therapy regulatory pathway development
The FDA's OTAT division reviews gene therapy Investigational New Drug (IND) applications and Biologics License Applications (BLA) for CF and other genetic diseases. Recent guidance documents establish frameworks for:
Regulatory pathways available:
Regenerative Medicine Advanced Therapy (RMAT) designation for CF gene therapies demonstrating preliminary clinical evidence
Accelerated approval pathway based on lung function endpoints (FEV1)
Rare pediatric disease voucher eligibility
Expanded access protocols for patients with progressive disease
Safety requirements:
Biodistribution studies documenting vector targeting
Toxicology evaluations in relevant animal models
Immunogenicity monitoring protocols
Long-term follow-up plans (typically 15 years post-dosing)
How Nome helps: FDA guidance exists, but few families or clinicians know how to apply it to individualized CF gene therapies. Nome manages the entire approval process—preparing IND submissions, implementing safety monitoring per FDA specifications, coordinating with reviewers, and obtaining permissions required before treatment. This regulatory expertise separates "theoretically possible" from "patient receives therapy."
11. Academic Research Labs Pioneering Preclinical CF Models
Leading institutions:
University of Iowa: Ferret CFTR knockout models replicating human lung disease pathology
Nationwide Children's Hospital: Primary airway epithelial cell culture systems for drug testing
Case Western Reserve University: Organoid platforms enabling patient-specific efficacy screening
Research contributions:
Animal models demonstrating gene editing can restore functional CFTR protein expression
Organoid systems predicting patient response to specific gene therapy approaches
Pharmacology endpoints validated for clinical translation
Safety biomarkers identifying potential adverse effects before human dosing
Publication example: Research demonstrating success rates exceeding 60% for preclinical gene editing in CF animal models provides evidence supporting clinical trial initiation.
How Nome helps: Academic labs generate foundational science; Nome's partnerships provide those labs with access to patient data, clinical networks, and operational support that accelerates discovery. Families benefit when Nome coordinates between research labs validating therapies and clinical teams delivering treatment—connecting the pieces that institutional silos separate.
12. AI and Data Scientists Enabling Personalized CF Therapy Design
Key applications:
Machine learning variant prediction: Algorithms classifying CFTR variants of uncertain significance (VUS) as amenable to gene therapy, gene editing, or modulator approaches
AlphaFold protein modeling: Predicting how CFTR mutations affect protein folding and identifying correction strategies
Literature mining tools: Natural language processing analyzing thousands of CF research papers to identify relevant mechanistic insights
Patient matching algorithms: Connecting individuals with rare CFTR mutations to others sharing variants for data aggregation
Platform examples:
Clinical decision support systems ranking therapeutic options by evidence strength
Database integration tools synthesizing genetic, phenotypic, and outcomes data
Trial eligibility screening algorithms automating patient-protocol matching
How Nome helps: AI represents Nome's core differentiator. While individual tools exist, Nome's platform integrates them systematically—analyzing genetic data, mining literature, matching patients to trials, coordinating manufacturers, and managing regulatory submissions. What takes clinicians months of manual search and coordination, Nome's AI completes in minutes. This isn't incremental improvement; it's solving the operational bottleneck preventing personalized CF gene therapy from scaling.
Making CF Gene Therapy Accessible: How Nome Helps
The challenge without Nome:
Families facing CF diagnoses with CFTR mutations unresponsive to modulators typically encounter:
Fragmented expertise: Genetic counselors, gene therapy researchers, contract manufacturers, FDA regulators—each requiring separate coordination
Unclear timelines: Academic labs may quote 2-5 years from genetic confirmation to potential therapy access
Cost opacity: Development expenses ranging from hundreds of thousands to millions with no upfront transparency
Geographic barriers: Leading experts concentrated at UCLA, Johns Hopkins, Imperial College—inaccessible to most families
Execution gaps: Treatment plans stall during handoffs between sequencing labs, therapy designers, safety testing vendors, and clinical trial sites
Nome's solution for CF gene therapy:
1. Free genetic evaluation:
Share CFTR mutation data through Nome's platform
AI-generated, expert-reviewed assessment determines whether gene therapy, gene editing, mRNA replacement, or other approaches fit the specific mutation
Transparent feasibility scoring based on peer-reviewed studies, clinical trial data, and mechanistic rationale
2. Execution-ready action plan:
Complete therapeutic roadmap identifying molecule design, delivery vector selection, safety studies required, manufacturing partners, and regulatory pathway
Transparent cost structure with milestone-based pricing
Timeline estimates based on real manufacturing lead times and approval processes
3. Expert network coordination:
Nome connects families to researchers like Dr. Kohn at UCLA, Dr. Alton's UK consortium, Johns Hopkins clinical teams, or appropriate trial sites
Manages pre-clinical work across contract manufacturers
Eliminates the coordination burden that derails families navigating institutional bureaucracy and vendor relationships
4. Regulatory and safety management:
Pre-clinical safety testing compliant with FDA gene therapy guidance
IND preparation and submission support
Clinical monitoring protocols implementation
Ongoing communication with regulatory reviewers
5. Treatment delivery and longitudinal support:
Coordinate therapy administration with qualified clinical sites
"We're here" every step of the way—ongoing monitoring and management through treatment course
Connect families to additional support resources as needs evolve
Why CF families choose Nome:
Speed: 30-day therapeutic plans vs. 6-12 month academic research timelines
Transparency: Upfront cost structures vs. uncertain grant funding and institutional billing
Access: Virtual platform enabling families anywhere to receive expert-reviewed assessments without traveling to UCLA or London
AI advantage: Analyzes dozens of papers and databases on CFTR mutations in minutes instead of months
End-to-end coordination: From genetic evaluation through treatment delivery—Nome eliminates handoff failures between disconnected institutions
Context from research: With gene therapy participation increasingly recommended for eligible CF patients, demand exceeds academic research capacity. Nome scales access by systematizing what institutions handle manually—turning "intake chaos" into development-ready assessments using proven AI and operational automation.
Conclusion: From CF Diagnosis to Gene Therapy with Nome
The 12 experts and institutions documented here represent the cutting edge of cystic fibrosis gene therapy—from UCLA's inhalable CRISPR nanoparticles to Oxford's clinical trials, from CF Foundation's $24 million investment in Prime Medicine to FDA's regulatory frameworks enabling personalized therapies. They've advanced gene therapy for CF patients with null mutations and other variants unresponsive to modulator drugs.
But expertise alone doesn't deliver treatment. The 10-20% of CF patients who cannot benefit from current drugs need coordination, speed, transparency, and systematic execution connecting research breakthroughs to bedside reality.
That's where Nome transforms potential into treatment:
AI-powered analysis evaluates CFTR mutations against all available therapeutic approaches in minutes
Expert-reviewed assessments provide families with prioritized options, evidence citations, and feasibility scores
Operational coordination connects families to researchers like Dr. Kohn, trial sites like Johns Hopkins, and contract manufacturers producing AAV vectors—with Nome managing execution
Regulatory expertise navigates FDA submissions and safety approvals that few families or clinicians understand
Transparent pricing makes gene therapy development financially predictable instead of prohibitively uncertain
For CF families post-diagnosis, time matters. Every month spent coordinating vendors, interpreting research papers, and managing institutional handoffs is a month of lung function decline. Nome eliminates that burden—managing the process at every stage so families can focus on what matters: getting their child treatment.
The experts in this directory built the scientific foundation. Nome built the system that gets CF patients treated.
Share your genetic diagnosis at nome.bio to receive a free AI-generated, expert-reviewed report scoring whether personalized gene therapy is possible for your CFTR mutation—and the clear next steps to make it happen.
