Cystic fibrosis is a progressive inherited genetic disorder that affects the lungs, pancreas, and other organs. It is caused by mutations in the CFTR gene that lead to abnormal ion transport and thick, sticky mucus in multiple organs. The disease is chronic and requires long-term multidisciplinary management.
Vertex secured a Germany reimbursement agreement for CASGEVY and signed a WuXi Biologics pact for a preclinical autoimmune T-cell engager. The moves add to efforts to build revenue beyond cystic fibrosis.
Vertex completed the rolling BLA submission for povetacicept in IgA nephropathy after positive Phase 3 RAINIER interim data. The company plans to use a Priority Review Voucher to accelerate FDA review.
Krystal Biotech said IOLITE enrollment for KB803 is complete and it remains on track for KB803 and KB801 registrational data readouts in 2026. The company also reported FDA platform technology designations for KB407 and KB111 and continued VYJUVEK expansion.
Researchers developed a lipid nanoparticle gene-editing approach that inserted a full CFTR gene into airway cells and restored 88% to 100% of normal channel function in a cystic fibrosis lab model.
Eli Lilly, AbbVie, Vertex, Gilead Sciences and Pfizer were identified as pharmaceutical stocks to watch, with Abbott Laboratories and McKesson appearing on a separate seven-stock list.
A lipid nanoparticle gene-editing approach inserted a full healthy CFTR gene into human airway cells and restored 88% to 100% of normal CFTR function in lab tests. The nonviral strategy is intended as a mutation-agnostic path for cystic fibrosis.
India's rare disease treatment funding fell to Rs 32.73 crore in 2025-26 from Rs 82.87 crore a year earlier, while several Centres of Excellence reported unspent balances. In Madhya Pradesh, concerns were also raised over the absence of dedicated rare disease centres in most state-run medical colleges.
CRISPR Therapeutics reported a $581.6 million net loss in 2025 with revenue of just $3.5 million, while continuing to advance its gene-editing pipeline including the approved CASGEVY therapy and next-generation CAR T cell programs. The company maintains strategic partnerships and expects current funds to support operations for at least 24 months.
Krystal Biotech is set to report fourth-quarter results with analysts expecting $1.58 a share on $106.16 million in revenue. Investors are watching Vyjuvek growth and pipeline programs including KB801, KB407 and KB707.
The global orphan drugs market is projected to grow from $223.76 billion in 2023 to $486.51 billion by 2032, with North America leading at 38% market share. Over 500 orphan drugs are approved with more than 800 candidates in clinical trials, driven by regulatory incentives and major pharmaceutical company investments in rare disease therapies.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07519460 |
Effects of High-Frequency Chest Wall Oscillation Combined With Concurrent Training on Cough,in Cystic Fibrosis Patients |
NOT_YET_RECRUITING | NA |
| NCT07500493 |
Tele-Rehabilitation in Adults With Cystic Fibrosis |
NOT_YET_RECRUITING | NA |
| NCT07485543 |
Safety and Efficacy of Inhaled BMD003 (CFTR mRNA) in Chinese Cystic Fibrosis Patients Aged ≥12 Years |
NOT_YET_RECRUITING | EARLY_PHASE1 |
| NCT07437105 |
Dose Escalation Study Evaluating the Safety and Pharmacokinetics of VX-272 in Healthy Participants |
RECRUITING | PHASE1 |
| NCT07414810 |
Novel Point-of-care Sweat Chloride Testing Device for Monitoring CFTR Function |
NOT_YET_RECRUITING | NA |
| NCT07349394 |
Effect of Vanzacaftor/Tezacaftor/Deutivacaftor (VNZ/TEZ/D-IVA) on the PK of Rosuvastatin in Healthy Participants |
COMPLETED | PHASE1 |
| NCT07342920 |
Study of the Prevalence of Active/Passive Smoking and Vaping Among Patients With Cystic Fibrosis |
RECRUITING | |
| NCT07283770 |
Dose Escalation Study Evaluating the Safety and Pharmacokinetics of VX-581 in Healthy Participants |
RECRUITING | PHASE1 |
| NCT07274631 |
A Cohort for Inflammatory Respiratory Diseases: From Phenotyping to Personalised Medicine |
RECRUITING | |
| NCT07088614 |
Effectiveness of Respiratory Physiotherapy on Frequency of Exacerbations, Lung Function and Quality of Life After 6 Months in Children With Cystic Fibrosis |
COMPLETED | NA |
