Table 1.
Biological therapy medicines approved or in an approval process in the European Union and medicines at a preclinical state in the European Union and the United States of America. The medicines in this table depict a part of the biological therapy treatments that are approved or in preclinical state. More information about these treatments and their state could be found in https://crisprmedicinenews.com/, https://www.ema.europa.eu/en, https://clinicaltrials.gov/ct2/home.
| Approved or in approval process (in European Union) | |||||
|---|---|---|---|---|---|
| Disease | Treatment target | Therapy type | Medicine name | State of the medicine | Source of information about the medicine |
| Hereditary diseases | |||||
| Metachromatic leukodystrophy | ARSA gene | Gene therapy | Libmeldy | Authorised (approved) | https://www.ema.europa.eu/en/medicines/human/EPAR/libmeldy |
| Severe combined immunodeficiency due to ADA deficiency | ADA gene | Gene therapy | Strimvelis | Authorised (approved) | https://www.ema.europa.eu/en/medicines/human/EPAR/strimvelis |
| Inherited retinal dystrophy (retinitis pigmentosa) | RPE65 gene | Gene therapy | Luxturna | Additional monitoring | https://www.ema.europa.eu/en/medicines/human/EPAR/luxturna |
| Hereditary transthyretin amyloidosis | TTR gene | Antisense therapy | Tegsedi | Authorised (approved) | https://www.ema.europa.eu/en/medicines/human/EPAR/tegsedi |
| Acute hepatic porphyria | ALAD gene | Antisense therapy | Givlaari | Authorised (approved) | https://www.ema.europa.eu/en/medicines/human/EPAR/givlaari |
| Spinal muscular atrophy (type 1, 2 and 3) | SMN2 gene | Antisense therapy | Evrysdi | Authorised (approved) | https://www.ema.europa.eu/en/medicines/human/EPAR/evrysdi |
| Spinal muscular atrophy (type 1) | SMN1 gene | Gene therapy | Zolgensma | Conditional approval | https://www.ema.europa.eu/en/medicines/human/EPAR/zolgensma |
| Beta thalassaemia | HBB gene | Gene therapy | Zynteglo | Under evaluation by EMA | https://www.ema.europa.eu/en/medicines/human/referrals/szynteglo |
| Early cerebral adrenoleukodystrophy | ABCD1 gene | Gene therapy | Skysona | Recommendation for EMA to grant a marketing authorisation | https://www.ema.europa.eu/en/medicines/human/summaries-opinion/skysona |
| Malignancies | |||||
| Diffuse large B-cell lymphoma, primary mediastinal large B-cell lymphoma | Gene encoding CAR protein | Gene therapy | Yescarta | Authorised (approved) | https://www.ema.europa.eu/en/medicines/human/EPAR/yescarta |
| B-cell acute lymphoblastic leukaemia, diffuse large B-cell lymphoma | Gene encoding CAR protein | Gene therapy | Kymriah | Authorised (approved) | https://www.ema.europa.eu/en/medicines/human/EPAR/kymriah |
| Preclinical state (in European Union, United States of America) | |||||
| Disease | Treatment target | Therapy type | Medicine name | State of the medicine | Source of information about the medicine |
| Hereditary diseases | |||||
| Mucopolysaccharidosis (type 1) | IDUA gene | Gene editing (Zinc finger nuclease) | SB-318 | Active clinical trial, not recruiting potential participants yet | https://clinicaltrials.gov/ct2/show/NCT02702115?term=NCT027021 15&draw=2&rank=1 https://crisprmedicinenews.com/clinical-trial/mucopolysaccharidosis-type-i-mps-i-nct02702115/ |
| Mucopolysaccharidosis (type 2) | IDS gene | Gene editing (Zinc finger nuclease) | SB-913 | Active clinical trial, not recruiting potential participants yet | https://clinicaltrials.gov/ct2/show/NCT03041324?term=gene+editing&recrs=d&draw=2&rank=3 https://crisprmedicinenews.com/clinical-trial/mucopolysaccharidosis-ii-mps-ii-nct03041324/ |
| Transfusion dependent Beta-Thalassemia, Sickle Cell Disease | BCL11A gene | Geneediting (CRISPR-Cas | CTX001 | Recruiting participants | https://clinicaltrials.gov/ct2/show/NCT03655678?term=CTX001&draw=2&rank=3 https://clinicaltrials.gov/ct2/show/NCT03745287?term=CTX001&dr aw=2&rank=2 |
| Leber Congenital Amaurosis (Type 10) | CEP290 gene | Gene editing (CRISPR-Cas9) | EDIT-101 | Recruiting participants | https://clinicaltrials.gov/ct2/show/NCT03872479?term=EDIT-101&draw=2&rank=1 https://crisprmedicinenews.com/clinical-trial/leber-congenital-amaurosis-nct03872479/ |
| Hereditary Transthyretin Amyloidosis | TTR gene | Gene editing (CRISPR-Cas9) | NTLA-2001 | Recruiting participants | https://clinicaltrials.gov/ct2/show/NCT04601051?term=NCT046010 51&draw=2&rank=1 https://crisprmedicinenews.com/clinical-trial/transthyretin-amyloidosis-attr-nct04601051/ |
| Infectious diseases | |||||
| Refractory herpetic viral keratitis | Herpes simplex virus type I genome | Gene editing (CRISPR-Cas9) | BD111 | Active clinical trial, not recruiting potential participants yet | https://clinicaltrials.gov/ct2/show/NCT04560790?term=gene+editing &draw=2&rank=1 https://crisprmedicinenews.com/clinical-trial/herpes-simplex-virus-refractory-keratitis-nct04560790/ |
| Human Immunodeficiency Virus Infection | CCR5 gene | Gene editing (CRISPR-Cas9) | CCR5 gene modification | Unknown (A study on whose the status has not been last verified within the past 2 years) | https://clinicaltrials.gov/ct2/show/NCT03164135?term=NCT03164135&draw=2&rank=1 https://crisprmedicinenews.com/clinical-trial/human-immunodeficiency-virus-infection-hiv-nct03164135/ |
| Malignancies | |||||
| Relapsed or refractory renal cell carcinoma | TRAC locus | Gene editing (CRISPR-Cas9) | CTX130 | Recruiting participants | https://clinicaltrials.gov/ct2/show/NCT04438083?term=gene+editing&recrs=a&draw=2&rank=9 https://crisprmedicinenews.com/ clinical-trial/renal-cell-carcinoma-rcc-nct04438083/ |
| Gastro-Intestinal Cancer | CISH gene | Gene editing (CRIPSR-Cas9) | TumorInfiltrating Lymphocytes (TIL) | Recruiting participants | https://clinicaltrials.gov/ct2/show/NCT04426669?term=gene+editing &draw=2&rank=8 https://crisprmedicinenews.com/clinical-trial/gastro-intestinal-cancer-gi-nct04426669/ |
| Human Papillomavirus- Related Malignant Neoplasm | Human papillomavirus genes encoding proteins E6 and E7 | Gene editing (TALENs) | T27 and T512 | Recruiting participants | https://clinicaltrials.gov/ct2/show/NCT03226470?term=NCT03226470&draw=2&rank=1 https://crisprmedicinenews.com/clinical-trial/human-papillomavirus-hpv-related-cervical-cancer-nct03226470/ |
| Non-Hodgkin Lymphoma | TRAC locus | Gene editing (meganuclease) | PBCAR19B | Recruiting participants | https://clinicaltrials.gov/ct2/show/study/NCT04649112 https://investor.precisionbiosciences.com/news-releases/news-release-details/precision-biosciences-receives-notice-allowance-us-patent https://crisprmedicinenews.com/clinical-trial/haematologic-malignancy-non-hodgkin-lymphoma-nhl-nct04649112/ |
| Metastatic Non-small Cell Lung Cancer | PDCD1 gene | Gene editing (CRISPR-Cas9) | PD-1 Knockout T Cells | Completed | https://clinicaltrials.gov/ct2/show/NCT02793856?term=NCT027938 56&draw=2&rank=1 https://crisprmedicinenews.com/clinical-trial/metastatic-non-small-cell-lung-cancer-nsclc-nct02793856/ |