Medicine library

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Some records contain detailed evidence; others currently contain only basic identity and regulatory information. Reviewed conclusions appear only when they are available for a specific use and group of people.

Showing 1–24 of 24 records

Exagamglogene autotemcel (Casgevy)
Gene-editing or gene therapyApproved in the United States (FDA)Detailed record
Use listed on the medicine record
Sickle cell disease and transfusion-dependent beta-thalassemia
Lovotibeglogene autotemcel (Lyfgenia)
Gene-editing or gene therapyApproved in the United States (FDA)Detailed record
Use listed on the medicine record
Sickle cell disease with a history of severe pain crises
Onasemnogene abeparvovec (Zolgensma)
Gene-editing or gene therapyApproved in the United States (FDA)Detailed record
Use listed on the medicine record
Spinal muscular atrophy in babies under two
Voretigene neparvovec (Luxturna)
Gene-editing or gene therapyApproved in the United States (FDA)Detailed record
Use listed on the medicine record
An inherited form of childhood blindness caused by two faulty RPE65 genes
Etranacogene dezaparvovec (Hemgenix)
Gene-editing or gene therapyApproved in the United States (FDA)Detailed record
Use listed on the medicine record
Haemophilia B in adults
Atidarsagene Autotemcel (Lenmeldy)
Gene-editing or gene therapyLaboratory or animal-stage researchExpanded record
Use listed on the medicine record
LENMELDY is indicated for the treatment of children with pre-symptomatic late infantile (PSLI), pre-symptomatic early juvenile (PSEJ) or early symptomatic early juvenile (ESEJ) metachromatic leukodystrophy (MLD). LENMELDY is an autologous hematopoietic stem cell-based gene therapyindicated for the treatment of children with pre-symptomatic late infantile (PSLI), pre-symptomatic early juvenile (PSEJ) or early symptomatic early juvenile (ESEJ) metachromatic leukodystrophy (MLD).
Eladocagene Exuparvovec (Kebilidi)
Gene-editing or gene therapyLaboratory or animal-stage researchExpanded record
Use listed on the medicine record
Aromatic L-amino acid decarboxylase (AADC) deficiency
Marnetegragene Autotemcel (Kresladi)
Gene-editing or gene therapyLaboratory or animal-stage researchExpanded record
Use listed on the medicine record
KRESLADI is indicated for the treatment of pediatric patients with severe leukocyte adhesion deficiency-I (LAD-I) due to biallelic variants in ITGB2 without an available human leukocyte antigen (HLA)-matched sibling donor for allogeneic hematopoietic stem cell transplant. This indication is approved under accelerated approval based on increase in neutrophil CD18 and CD11a surface expression [see Clinical Studies (14) ]. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).
Tisagenlecleucel (Kymriah)
Gene-editing or gene therapyLaboratory or animal-stage researchExpanded record
Use listed on the medicine record
KYMRIAH is a CD19-directed genetically modified autologous T cell immunotherapy indicated for the treatment of: Patients up to 25 years of age with B-cell precursor acute lymphoblastic leukemia (ALL) that is refractory or in second or later relapse. ( 1.1 ) Adult patients with relapsed or refractory (r/r) large B-cell lymphoma after two or more lines of systemic therapy, including diffuse large B-cell lymphoma (DLBCL) not otherwise specified, high grade B-cell lymphoma and DLBCL arising from follicular lymphoma.
Zopapogene Imadenovec (Papzimeos)
Gene-editing or gene therapyLaboratory or animal-stage researchExpanded record
Use listed on the medicine record
Recurrent respiratory papillomatosis
Axicabtagene Ciloleucel (Yescarta)
Gene-editing or gene therapyLaboratory or animal-stage researchExpanded record
Use listed on the medicine record
YESCARTA is a CD19-directed genetically modified autologous T cell immunotherapy indicated for the treatment of: Adult patients with large B-cell lymphoma that is refractory to first-line chemoimmunotherapy or that relapses within 12 months of first-line chemoimmunotherapy. ( 1.1 ) Adult patients with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy, including diffuse large B-cell lymphoma (DLBCL) not otherwise specified, primary mediastinal large B-cell lymphoma, high grade B-cell lymphoma, and DLBCL arising from follicular lymphoma.
Donislecel (Lantidra)
Gene-editing or gene therapyLaboratory or animal-stage researchExpanded record
Use listed on the medicine record
LANTIDRA is an allogeneic pancreatic islet cellular therapy indicated for the treatment of adults with Type 1 diabetes who are unable to approach target HbA1c because of current repeated episodes of severe hypoglycemia despite intensive diabetes management and education. Use LANTIDRA in conjunction with concomitant immunosuppression.
Etuvetidigene Autotemcel (Waskyra)
Gene-editing or gene therapyLaboratory or animal-stage researchExpanded record
Use listed on the medicine record
WASKYRA ® is indicated for the treatment of pediatric patients aged 6 months and older and adults with Wiskott-Aldrich Syndrome (WAS) who have a mutation in the WAS gene and for whom hematopoietic stem cell transplantation (HSCT) is appropriate and no suitable human leukocyte antigen (HLA)-matched related stem cell donor is available.
Lunsotogene Parvec (Otarmeni)
Gene-editing or gene therapyLaboratory or animal-stage researchExpanded record
Use listed on the medicine record
OTARMENI is indicated for the treatment of pediatric and adult patients with severe-to-profound and profound sensorineural hearing loss (any frequency >90 dB HL) associated with molecularly confirmed biallelic variants in the OTOF gene, preserved outer hair cell function, and no prior cochlear implant in the same ear. This indication is approved under accelerated approval based on the improvement of hearing sensitivity assessed by average pure tone audiometry (PTA) at Week 24 [see Clinical Studies (14) ] .
Afamitresgene Autoleucel (Tecelra)
Gene-editing or gene therapyLaboratory or animal-stage researchExpanded record
Use listed on the medicine record
Unresectable or metastatic synovial sarcoma who have received prior chemotherapy, are HLA-A*02
Ciltacabtagene Autoleucel (Carvykti)
Gene-editing or gene therapyLaboratory or animal-stage researchExpanded record
Use listed on the medicine record
CARVYKTI is indicated for the treatment of adult patients with relapsed or refractory multiple myeloma, who have received at least 1 prior line of therapy, including a proteasome inhibitor and an immunomodulatory agent, and are refractory to lenalidomide. CARVYKTI is a B-cell maturation antigen (BCMA)-directed genetically modified autologous T cell immunotherapy indicated for the treatment of adult patients with relapsed or refractory multiple myeloma who have received at least 1 prior line of therapy, including a proteasome inhibitor and an immunomodulatory agent, and are refractory to…
Prademagene Zamikeracel (Zevaskyn)
Gene-editing or gene therapyLaboratory or animal-stage researchExpanded record
Use listed on the medicine record
Wounds
Betibeglogene Autotemcel (Zynteglo)
Gene-editing or gene therapyLaboratory or animal-stage researchExpanded record
Use listed on the medicine record
Β-thalassemia who require regular red blood cell (RBC) transfusions
Brexucabtagene Autoleucel (Tecartus)
Gene-editing or gene therapyLaboratory or animal-stage researchExpanded record
Use listed on the medicine record
Relapsed or refractory mantle cell lymphoma
Lifileucel (Amtagvi)
Gene-editing or gene therapyLaboratory or animal-stage researchExpanded record
Use listed on the medicine record
Unresectable or metastatic melanoma previously treated with a PD-1 blocking antibody, and if BRAF V600 mutation positive
Talimogene Laherparepvec (Imlygic)
Gene-editing or gene therapyLaboratory or animal-stage researchExpanded record
Use listed on the medicine record
The local treatment of unresectable cutaneous, subcutaneous, and nodal lesions
Elivaldogene Autotemcel (Skysona)
Gene-editing or gene therapyLaboratory or animal-stage researchExpanded record
Use listed on the medicine record
SKYSONA is indicated to slow the progression of neurologic dysfunction in boys 4-17 years of age with early, active cerebral adrenoleukodystrophy (CALD) without an available human leukocyte antigen (HLA)-matched donor for allogeneic hematopoietic stem cell transplant. Early, active cerebral adrenoleukodystrophy refers to asymptomatic or mildly symptomatic (neurologic function score, NFS ≤ 1) boys who have gadolinium enhancement on brain magnetic resonance imaging (MRI) and Loes scores of 0.5-9.
Idecabtagene Vicleucel (Abecma)
Gene-editing or gene therapyLaboratory or animal-stage researchExpanded record
Use listed on the medicine record
Relapsed or refractory multiple myeloma after two or more prior lines of therapy including an immunomodulatory agent
Nadofaragene Firadenovec (Adstiladrin)
Gene-editing or gene therapyLaboratory or animal-stage researchExpanded record
Use listed on the medicine record
High-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-Muscle Invasive Bladder Cancer (NMIBC) with carcinoma in situ

Exagamglogene autotemcel (Casgevy)

Lovotibeglogene autotemcel (Lyfgenia)

Onasemnogene abeparvovec (Zolgensma)

Voretigene neparvovec (Luxturna)

Etranacogene dezaparvovec (Hemgenix)

Atidarsagene Autotemcel (Lenmeldy)

Eladocagene Exuparvovec (Kebilidi)

Marnetegragene Autotemcel (Kresladi)

Tisagenlecleucel (Kymriah)

Zopapogene Imadenovec (Papzimeos)

Axicabtagene Ciloleucel (Yescarta)

Donislecel (Lantidra)

Etuvetidigene Autotemcel (Waskyra)

Lunsotogene Parvec (Otarmeni)

Afamitresgene Autoleucel (Tecelra)

Ciltacabtagene Autoleucel (Carvykti)

Prademagene Zamikeracel (Zevaskyn)

Betibeglogene Autotemcel (Zynteglo)

Brexucabtagene Autoleucel (Tecartus)

Lifileucel (Amtagvi)

Talimogene Laherparepvec (Imlygic)

Elivaldogene Autotemcel (Skysona)

Idecabtagene Vicleucel (Abecma)

Nadofaragene Firadenovec (Adstiladrin)