Cell and Gene Therapy Development for Rare Lung Diseases
Patients with rare pulmonary disorders have endured decades of therapeutic stagnation, constrained by the limitations of conventional small-molecule approaches and the biological complexity of respiratory pathologies. Cell and gene therapies (CGT) now represent a paradigm shift, offering the first genuine opportunities for disease modification rather than symptomatic management in conditions like idiopathic pulmonary fibrosis, cystic fibrosis, and alpha-1 antititrypsin deficiency.
Our vertically integrated CGT development platform empowers innovators to overcome the unique challenges of pulmonary therapeutics:
- Biologic delivery barriers (mucociliary clearance, alveolar macrophages, air-blood interface)
- Disease heterogeneity across >200 classified rare lung disorders
- Regulatory complexities for first-in-class pulmonary biologics
We're not just CRO providers-we're development partners committed to transforming pulmonary medicine by accelerating your program's path to regulatory approval and patient impact.
Challenges & Therapeutic Potential
Current Limitations & Unmet Needs
1. Limited Disease-Modifying Therapies
Approximately 80% of chronic lung diseases, including idiopathic pulmonary fibrosis (IPF) and cystic fibrosis (CF), lack FDA-approved treatments that target disease progression. Most existing therapies (e.g., corticosteroids, antifibrotics) focus on symptom management rather than addressing genetic or molecular root causes.
2. Poor Prognoses
Aggressive conditions like IPF have a median survival of 2–3 years post-diagnosis. Even with advancements in supportive care, mortality rates remain high due to irreversible tissue damage and limited regenerative capacity in lung tissue.
3. Systemic Side Effects
Conventional therapies often require systemic administration, increasing risks of off-target effects (e.g., immunosuppression, organ toxicity).
4. Delivery Challenges
The unique physiology of the lung poses significant barriers to effective therapy. Rapid epithelial turnover shortens the duration of therapeutic transgene expression, requiring frequent re-administration of treatments like inhaled CRISPR-based therapies. Immune responses against delivery vectors (e.g., AAV, lipid nanoparticles) or transgenes further compromise efficacy, particularly in patients with pre-existing immunity. The blood-air barrier limits systemic delivery while demanding precision in localized targeting to avoid systemic toxicity. Additionally, pre-existing lung dysfunction in diseases such as COPD or IPF necessitates rigorous safety assessments to prevent therapy-induced exacerbation of inflammation or fibrosis.
Why Cell and Gene Therapy (CGT)?
CGT addresses the limitations of traditional approaches by targeting disease mechanisms at their source. Gene replacement strategies, such as delivering functional CFTR genes to correct cystic fibrosis mutations, directly resolve the root cause of cellular dysfunction. Innovations in gene editing, including CRISPR-based platforms, enable precise correction of genetic defects like those in alpha-1 antitrypsin deficiency (AATD). Cell-based therapies, such as mesenchymal stem cells (MSCs), offer regenerative potential by modulating inflammation and promoting tissue repair in conditions like IPF. Additionally, advancements in targeted pulmonary delivery systems-such as inhaled viral vectors or lipid nanoparticles-ensure high local drug concentrations while minimizing systemic exposure.
Figure 1. Vector platforms and their characteristics available for gene transfer. (Bisserier M, et al., 2022)Our Services
Core Service Offerings
1. Target Identification & Lead Optimization
What We Do:
We identify disease targets and optimize therapeutic leads using gene editing, RNA therapies, and stem cell engineering, supported by AI-driven bioinformatics and 3D lung models.
Your Benefit:
- Faster lead selection with clinically relevant data.
- Reduced risk via multi-omics validation (genomics, proteomics).
2. Vector & Delivery System Design
What We Do:
We engineer lung-targeted delivery systems, including viral vectors (e.g., AAVs) and non-viral nanoparticles, and optimize inhalation devices with immune-evasion strategies to enhance treatment durability.
Your Benefit:
- Localized therapy with minimal systemic exposure.
- Enhanced durability of gene/cell therapies in lung tissue.
3. Preclinical & Translational Testing
What We Do:
We validate therapies in disease-specific models and humanized lung systems, generating GLP-compliant preclinical data packages to support IND submissions.
Your Benefit:
- IND-ready data packages aligned with FDA/EMA guidelines.
- Reduced clinical risk with human-relevant validation.
4. Scalable Manufacturing & Quality Control
What We Do:
We provide GMP-grade production of viral vectors and cell therapies, ensuring stability and batch consistency for inhalable formulations, with end-to-end quality control from preclinical to commercial stages.
Your Benefit:
- Seamless scaling from preclinical to commercial batches.
- Regulatory-compliant production to accelerate approvals.
Advanced Methodologies & Technologies
| Platform | Key Features |
|---|---|
| Lung-Targeted Delivery | AAV capsid engineering, nebulization optimization, mucus-penetrating formulations |
| Disease-Relevant Models | Patient-derived ALI cultures, lung-on-a-chip, iPSC-derived organoids |
| Gene Editing & Regulation | CRISPR, base/prime editing, epigenetic silencing, safe-harbor integration |
| Cell Therapy Production | GMP-compliant MSC/iPSC differentiation, immune cell engineering |
| Analytics & QC | Vector titering, cell product characterization, replication-competent virus testing |
Service Workflow
Why Choose Us?
- Deep rare lung disease expertise
- Specialized pulmonary delivery solutions
- Flexible small-batch GMP production
- Strong regulatory track record
FAQs?
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Q: What makes your preclinical models uniquely suited for rare lung diseases?
A: We employ patient-derived organoids, humanized murine models (e.g., NOG-EXL with engrafted airway epithelium), and precision large-animal models (CFTR-KO pigs). Our platforms replicate human disease pathophysiology better than standard models, with functional readouts like mucociliary clearance, TEER, and surfactant secretion for clinically relevant data.
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Q: How do you optimize gene/cell therapies for lung-specific delivery?
A: Our AAV capsid evolution platform screens for optimal bronchial/alveolar tropism, while nebulization/LNP formulation studies assess stability and deposition efficiency. We also test mucus-penetrating additives (e.g., PEGylation) and cell priming strategies (hypoxia conditioning for fibrotic niches).
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Q: Are your preclinical studies compliant with FDA/EMA IND requirements?
A: Yes. We conduct GLP toxicology (28-day repeat-dose in 2 species), biodistribution (QPCR-based vector tracking), and reproductive toxicity screens. Our reports include gap analyses to align with orphan drug/Breakthrough Therapy designation pathways.
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Q: How do you accelerate timelines without compromising data quality?
A: Through parallel processing (e.g., running GLP tox alongside biodistribution) and modular workflows. Example: Lead optimization (8–12 weeks) includes in vitro efficacy + preliminary stability testing simultaneously.
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Q: What happens after preclinical studies? Do you assist with clinical transition?
A: We provide tech transfer packages (CMC documentation, SOPs, raw datasets) and partner introductions to specialized CMOs/CDMOs. While we don't conduct clinical trials, we bridge the gap with regulatory consultants and investor-ready data packaging.
Reference
- Bisserier M.; et al. Novel Insights into the Therapeutic Potential of Lung-Targeted Gene Transfer in the Most Common Respiratory Diseases. Cells. 2022;11(6):984.
All of our services and products are intended for preclinical research use only and cannot be used to diagnose, treat or manage patients.