Commercial-stage biotechnology company that discovers, develops, manufactures and commercializes genetic medicines for diseases with high unmet medical need.
Clinical Trials
19
4 active
Approvals
2
Total approvals
Agencies
1
Regulatory bodies
Founded
2015
Active, not recruiting
3
15.8%
Completed
4
21.1%
Not yet recruiting
1
5.3%
Recruiting
9
47.4%
Withdrawn
2
10.5%
- Over 30,000 people in the U.S. live with cystic fibrosis, with rising diagnosed prevalence driving demand for targeted therapies and precision medicine approaches. - CFTR modulator therapies have dramatically transformed treatment by targeting underlying CFTR gene mutations, improving lung function, life expectancy, and reducing hospital admissions. - Gene therapy and CRISPR-based gene-editing technologies are emerging as potential long-term or permanent solutions, though they remain in development. - High drug costs and limited treatment options for rare CFTR mutations continue to pose significant barriers to patient access globally.
- The UK Medicines and Healthcare products Regulatory Agency approved VYJUVEK (beremagene geperpavec-svdt) for treating wounds in dystrophic epidermolysis bullosa patients with COL7A1 gene mutations from birth. - VYJUVEK becomes the first genetic medicine approved in the UK for DEB treatment, offering flexible administration options including home dosing by patients or caregivers. - Clinical trials demonstrated 67% complete wound healing at six months compared to 22% for placebo in patients aged one to 44 years. - The approval grants VYJUVEK orphan designation with up to 12 years of market exclusivity in the UK, following previous approvals in the US, EU, and Japan.
- Moderna expects phase 3 data for norovirus and adjuvant melanoma programs in 2026, with the company up 82% year-to-date following a $2.25 billion patent settlement. - Krystal Biotech demonstrates strong fundamentals with 94% gross margins and $204 million net income in 2025, while advancing phase 3 trials in corneal DEB and neurotrophic keratitis. - Sarepta Therapeutics faces recovery challenges after ELEVIDYS revenue dropped 33% year-over-year due to safety-driven shipment suspensions, though management targets profitability return in 2026. - FDA regulatory environment under new leadership and potential staffing reductions could significantly impact approval timelines across the biotech sector.
- ACT Capital Management established a new $8 million position in TG Therapeutics, acquiring 268,875 shares despite the biotech's 30% stock decline over the past year. - TG Therapeutics' multiple sclerosis drug BRIUMVI generated approximately $606.9 million in product sales in 2025, nearly doubling year-over-year and driving total revenue to $616.3 million. - The company projects $875-900 million in total global revenue for 2026, driven by continued BRIUMVI adoption and international expansion efforts. - The investment represents 6.5% of ACT Capital's $123.67 million portfolio, positioning alongside other healthcare and biotech holdings including Krystal Biotech and Abivax.
- The FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation to KB707, Krystal Biotech's inhaled immunotherapy targeting advanced or metastatic non-small cell lung cancer. - KB707 is designed as a redosable gene therapy that drives sustained, localized expression of interleukin-2 and interleukin-12 in the tumor microenvironment. - Early clinical evidence from the ongoing KYANITE-1 study demonstrated consistent antitumor activity, including durable responses and clinically significant tumor reductions in heavily pre-treated patients. - The RMAT designation provides accelerated development benefits including rolling review, intensive FDA guidance, and potential for accelerated approval pathways.
- Krystal Biotech will announce interim clinical results from the highest dose cohort of its Phase 1 CORAL-1 study evaluating KB407 gene therapy in cystic fibrosis patients on January 8, 2026. - The update will include molecular assessments of KB407 transduction and wild-type CFTR protein expression following inhaled administration of the investigational therapy. - The CORAL-1 study is a multi-center, dose escalation Phase 1 trial designed to evaluate the safety and efficacy of KB407 in patients with cystic fibrosis.
- The FDA approved five new indications for previously approved dermatology drugs in 2025, including two additional approvals for Dupixent, bringing its total approved indications to eight diseases. - Zoryve (roflumilast) received expanded approvals for both cream formulation in young children with atopic dermatitis and foam formulation for plaque psoriasis in adolescents and adults. - Opzelura (ruxolitinib) became the first topical JAK inhibitor approved for pediatric atopic dermatitis patients ages 2-11 years in the United States. - Libtayo (cemiplimab) gained approval as the first immunotherapy for adjuvant treatment of high-risk cutaneous squamous cell carcinoma following surgery and radiation.
- The global cell and gene therapy market is expected to exceed $105 billion by 2033, driven by significant expansion in clinical pipelines with Phase I trials experiencing approximately 11% growth. - Cell therapy dominates the market with 61.2% share, reaching $14.66 billion in 2023, while manufacturing costs remain a challenge with CAR-T therapies exceeding $100,000 per patient. - North America leads the market with 43.7% share at $11.97 billion in 2023, supported by advanced regulatory frameworks including the FDA's RMAT designation for accelerated therapy review. - Advanced delivery vectors and viral vector platforms are driving innovation, with companies like AGC Biologics and Charles River Laboratories launching new manufacturing solutions to improve scalability and reduce costs.
- Krystal Biotech received FDA platform technology designation for its genetically modified HSV-1 viral vector used in KB801 gene therapy for neurotrophic keratitis. - The designation recognizes the reproducibility and scalability of Krystal's HSV-1 gene delivery platform and may provide development efficiencies. - KB801 is designed to enable sustained nerve growth factor expression in the eye, potentially reducing treatment burden compared to current six-times-daily NGF drops. - The company can leverage manufacturing and safety data from its FDA-approved VYJUVEK gene therapy to accelerate pipeline development.
- French biotech EG 427 reported that its HSV-1 gene therapy EG110A reduced urinary incontinence episodes by over 88% at week 12 in patients with neurogenic detrusor overactivity. - The therapy targets type C sensory neurons responsible for bladder muscle overactivity while preserving other bladder controls through a single treatment course of intradetrusor injections. - The positive results validate EG 427's HERMES nrHSV-1 technology platform and may enable expansion into other pain-related pathologies driven by type C sensory neuron activity. - NDO affects at least two million patients across seven major markets and can lead to death in 5-10% of spinal cord injury patients due to complications.
