Disease & Gene Tracking
Select sickle cell, thalassemia, SMA, Duchenne MD or CRISPR-Cas9 research — create your personalized news feed and notifications.
CRISPR, Gene Therapy &
Track Clinical Trials.
Monitor clinical trials, FDA approvals, and phase transitions in real-time for sickle cell disease, SMA, thalassemia, and 7,000+ rare genetic diseases.
CRISPR-Cas9Sickle CellSMA · ZolgensmaHereditary Hearing LossHemophilia
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From CRISPR to AAV vectors, Phase 1 to FDA approval — track the entire gene therapy process on one platform.
Clinical Trial Timeline
Track which gene therapy is at which stage from Phase 1 to FDA approval with live charts and up-to-date clinical trial data.
FDA & EMA Approval Alerts
Be the first to know when a new clinical trial starts, FDA or EMA approval arrives, or Phase 3 results are published.
Featured Diseases in Gene Therapy
Diseases with active clinical trials in CRISPR, AAV vector, and gene editing therapies — 372
#1Approved (Strimvelis)
ADA-SCID
ADA · Ex Vivo Gene Therapy
ADA
Affected~500
Immunologic#2Approved (Casgevy (exagamglogene autotemcel))
Beta-Thalassemia
HBB · CRISPR-Cas9
HBB
Affected~5M
Hemoglobin#3Approved (Hemgenix (etranacogene dezaparvovec))
Hemophilia B
F9 · AAV Gene Therapy
F9
Affected~30K
Hematologic#4Approved (Luxturna (voretigene neparvovec))
Leber Congenital Amaurosis
RPE65 · AAV Gene Therapy
RPE65
Affected~100K
Eye#5Approved (Casgevy (exagamglogene autotemcel))
Sickle Cell Disease
HBB · CRISPR-Cas9
HBB
Affected~7.7M
Hemoglobin#6Approved (Zolgensma (onasemnogene abeparvovec))
Spinal Muscular Atrophy
SMN1 · AAV Gene Therapy
SMN1
Affected~60K
Neuromuscular#7Research Stage
Severe Congenital Neutropenia
ELANE, HAX1
ELANEHAX1
Affected1/200000
Hematologic#8Research Stage
Maple Syrup Urine Disease
BCKDHA
BCKDHA
Affected1/185000
MetabolicHow It Works?
From CRISPR-Cas9 to AAV gene therapy, Phase 1 clinical trial to FDA approval — we help you track the entire process step by step.
Create Your Disease & Gene Profile
Select the disease or mutation you're interested in — sickle cell, SMA, hereditary hearing loss, Duchenne MD.
Monitor Clinical Trials in Real Time
Track 3,900+ active gene therapy trials by combining data from ClinicalTrials.gov, OMIM, and Orphanet.
Get Instant FDA, EMA & Phase Transition Alerts
Be the first to know when critical approvals like Casgevy, Zolgensma, or OTOF gene therapy are announced.
Example: CRISPR-Cas9 · Sickle Cell Disease (Casgevy)
FDA Approved — In Treatment Since 2023
Phase 1Phase 2Phase 3 ✓FDA ✓
3.900+
Active Clinical Trials
7.400+
Genetic Diseases
46
Countries Tracking
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