Clinical Trials
44
Trial Phases
3 Phases
Drug Approvals
10
Drug Approvals
Clinical Trials
Distribution across different clinical trial phases (28 trials with phase data)• Click on a phase to view related trials
News
Stoke Therapeutics Appoints Ian Smith as CEO as Phase 3 Trial for Dravet Syndrome Treatment Advances
Stoke Therapeutics has appointed Ian Smith as Chief Executive Officer after he served as Interim CEO since March 2025, bringing over 30 years of rare disease drug development experience.
Vertex Secures Italian Reimbursement for CRISPR Gene Therapy Casgevy, Expanding European Access for Blood Disorders
Vertex Pharmaceuticals announced a reimbursement agreement with Italy's Medicines Agency (AIFA) for Casgevy, the first CRISPR/Cas9 gene-edited therapy approved for transfusion-dependent beta thalassemia and sickle cell disease in Europe.
ToolGen Files Patent Infringement Lawsuit Against Lonza Over CRISPR Technology Used in CASGEVY Production
ToolGen has filed a patent infringement lawsuit in the Netherlands against Lonza, alleging unauthorized use of its CRISPR-Cas9 RNP technology in producing CASGEVY, the world's first approved gene-editing therapy.
WHO Adds GLP-1 Diabetes Drugs to Essential Medicines List, Excludes Weight-Loss Indication
The World Health Organization has added GLP-1 medications including semaglutide and tirzepatide to its essential medicines list for treating type 2 diabetes with comorbidities.
Vertex Pharmaceuticals Partners with Enlaza Therapeutics in $45 Million Deal to Develop Autoimmune and Gene-Editing Therapies
Vertex Pharmaceuticals has committed $45 million upfront to partner with Enlaza Therapeutics, with potential total deal value exceeding $2 billion for autoimmune disease treatments.
Kexing Biopharm Partners with IQVIA to Accelerate Global Drug Development and Market Access
Kexing Biopharm has entered a strategic partnership with IQVIA in August 2025 to accelerate global clinical development and regulatory approval of its innovative drugs and biosimilars, particularly targeting Europe and other regulated international markets.
Vertex Pharmaceuticals Discontinues VX-993 Development After Phase 2 Failure in Acute Pain
Vertex Pharmaceuticals announced that its experimental non-opioid pain reliever VX-993 failed to significantly outperform placebo in a Phase 2 trial for acute pain treatment.
Frazier Healthcare Partners Closes $1.3 Billion Fund for Early-Stage Biotech Investments
Frazier Healthcare Partners has successfully closed a $1.3 billion venture capital fund specifically targeting early-stage biotechnology companies and startup creation.
Vertex Pharmaceuticals Faces Setbacks in Pain Medicine Development Program
Vertex Pharmaceuticals disclosed two setbacks in its pain medicine development program, suggesting challenges in applying its iterative approach beyond cystic fibrosis treatments.
CMS Launches Multi-State Payment Model to Expand Access to Sickle Cell Gene Therapies
The Centers for Medicare & Medicaid Services unveiled a new outcomes-based payment model with 33 states to expand Medicaid access to gene therapies for sickle cell disease.