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Gene therapy
Gene therapy is a medical technology for treating or preventing disease by correcting the underlying genetic problem. Gene therapy techniques allow the treatment of genetic-based disorders by altering the genes that cause diseases
5 Facts you should know
FACT
Human gene therapy seeks to modify or manipulate the expression of a gene or to alter the biological properties of living cells for therapeutic use
FACT
Somatic gene therapy represents mainstream clinical research, in which a gene (therapeutic DNA) is used to treat disease
FACT
The first therapeutic use of gene transfer was performed by French Anderson in a trial starting in September 1990
FACT
In 2017, Spark Therapeutics' Luxturna for RPE65 mutation-induced blindness was the first therapeutic gene therapy approved in the United States
FACT
To date, most of the successful approaches in gene therapy utilize adeno-associated viruses (AAVs)
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Top Clinical Trials
Disease | Drug/Therapy | Development Phase | Company/Sponsor | Clinical Trials | More Information |
---|---|---|---|---|---|
Duchenne Muscular Dystrophy | PF-06939926 | Phase 3 | Pfizer | Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy | More Info |
Dystrophic Epidermolysis Bullosa | Beremagene Geperpavec | Phase 3 | Krystal Biotech | A Long-term Treatment With B-VEC for Dystrophic Epidermolysis Bullosa | More Info |
Glycogen storage disease (Type 1a) | DTX401 | Phase 3 | Ultragenyx | A Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients With Glycogen Storage Disease Type Ia (GSDIa) | More Info |
Hemophilia A | giroctocogene fitelparvovec | Phase 3 | Pfizer | Study to Evaluate the Efficacy and Safety of PF-07055480 / Giroctocogene Fitelparvovec Gene Therapy in Moderately Severe to Severe Hemophilia A Adults (AFFINE) | More Info |
Hemophilia A | valoctocogene Roxaparvovec | Phase 3 | BioMarin | Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients (BMN 270-301) (BMN 270-301 | More Info |
Hemophilia B | PF-06838435 | Phase 3 | Pfizer | A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B (BENEGENE-2) | More Info |
Hunter syndrome (MPS II) | RGX-121 | Phase 3 | Regenexbio | CAMPSIITE™ RGX-121 Gene Therapy in Subjects With MPS II (Hunter Syndrome) | More Info |
Mucopolysaccharidosis Type IIIA (MPS IIIA) | LYS-SAF302 | Phase2/3 | Lysogene | Study of AAVrh10-h.SGSH Gene Therapy in Patients With Mucopolysaccharidosis Type IIIA (MPS IIIA) (AAVance) | More Info |
Ornithine Transcarbamylase (OTC) Deficiency | DTX301 | Phase 3 | Ultragenyx | Clinical Study of DTX301 AAV- Mediated Gene Transfer for Ornithine Transcarbamylase(OTC) Deficiency | More Info |
Retinitis Pigmentosa | AAV5-RPGR 4e11 | Phase 3 | MeiraGTx UK II | Follow-up Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene | More Info |
Severe combined immunodeficiency syndrome | Blood cells transduced with the G2SCID vector | Phase 1/2 | Boston Children's Hospital | Phase I/II Trial of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan Conditioning | More Info |
Sickle cell disease | lovotibeglogene autotemcel | Phase 3 | bluebird bio | A Study Evaluating Gene Therapy With BB305 Lentiviral Vector in Sickle Cell Disease | More Info |
Spinal muscular atrophy | OAV-101 | Phase 3 | Novartis | Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA) (SMART) | More Info |
Wet AMD | RGX-314 | Phase 3 | Regenexbio | Pivotal 2 Study of RGX-314 Gene Therapy in Participants With nAMD (ASCENT) | More Info |
Wiskott-Aldrich Syndrome | OTL-103 | Phase 3 | Orchard Therapeutics | A Clinical Study to Evaluate the Use of a Cryopreserved Formulation of OTL-103 in Subjects With Wiskott-Aldrich Syndrome | More Info |
Current Approved Treatments
Product | Generic Name | Year Approved | Indications |
---|---|---|---|
Breyanzi | lisocabtagenemaraleucel | 2021 | Diffuse large B-cell lymphoma; follicular lymphoma |
Imlygic | talimogenelaherparepvec | 2015 | Melanoma |
Kymriah | tisagenlecleucel-t | 2017 | Acute lymphocytic leukemia; diffuse large B-cell lymphoma |
Luxturna | voretigeneneparvovec | 2017 | Leber’s congenital amaurosis |
Yescarta | axicabtageneciloleucel | 2017 | Diffuse large B-cell lymphoma; non-Hodgkin’s lymphoma; follicular lymphoma |
Zolgensma | onasemnogene abeparvovec | 2019 | Spinal muscular atrophy |
Zynteglo | betibeglogeneautotemcel | 2022 | Transfusion-dependent beta thalassemia |
Tecartus | brexucabtageneautoleucel | 2020 | Mantel cell lymphoma; acute lymphocytic leukemia |
Abecma | idecabtagenevicleucel | 2021 | Multiple myeloma |
Skysona | elivaldogene autotemcel | 2022 | Cerebral adrenoleukodystrophy (CALD) |
Carvykti | ciltacabtageneautoleucel | 2022 | Multiple myeloma |
Elevydis | delandistrogene moxeparvovec-rokl | 2023 | Duchenne muscular dystrophy (DMD) |
Hemgenix | etranacogene dezaparvovec-drlb | 2022 | Hemophilia B |
Roctavian | valoctocogene roxaparvovec-rvox | 2023 | Hemophilia A |
Vyjuvek | beremagene geperpavec | 2023 | Dystrophic epidermolysis bullosa |