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May 19, 2022 / eMedClub News/--Recently, Rocket Pharmaceutical announced its treatment of Fanconi Anemia (FA) at the 25th Annual Meeting of the American Society for Gene and Cell Therapy (ASGC.
Positive data from pivotal Phase 2 clinical trials and Phase 1 clinical trials in Danon Disease (DD) and Pyruvate Kinase Deficiency (PK.
➤ Ex vivo lentivirus-mediated gene therapy RP-L102 in patients with Fanconi anemia Fanconi anemia (FA) is a rare pediatric disease, and approximately 60-70% of FA patients have mutations in the FANCA gene, which encodes A protein essential for DNA repa.
Mutated genes in FANCA cause chromosome breakage and increase susceptibility to oxidative and environmental stre.
Patients are prone to bone marrow dysfunction and cancer susceptibili.
RP-L102 is a gene therapy that uses lentiviral vector to deliver normal FANCA gene to transform hematopoietic stem cells in vit.
The data presented here are from an initial 9 of 12 patients who received RP-L10 Five of the nine evaluable patients showed increased resistance of bone marrow cells to the DNA-damaging drug MMC, indicating an enhanced ability of patients' bone marrow cells to repair DNA damage, meeting the trial's primary endpoi.
Peripheral VCN demonstrated that the sixth patient showed evidence of progressively increasing gene correcti.
Two other patients are currently only 12 months after treatment, and one patient developed progressive bone marrow failure and underwent a successful allogeneic transpla.
Three of the 12 treated patients had not yet reached the 12-month time poi.
The safety profile of RP-L102 was favorable, with no evidence of dysplasia, clonal dominance, or oncogenic integration; as previously reported, one patient experienced a grade 2 transient infusion-related reaction that subsequently resolv.
➤ AAV9-mediated gene therapy RP-A501 for the treatment of Danon's disease Danon's disease (DD) is a very rare and life-threatening disease due to the failure of basic biological processes in cells responsible for the removal and recycling of protei.
This is a rare X-linked autophagic vacuolar myopathy that causes multiple organ dysfunction, including the heart, skeletal muscle, nervous system, eye, and liver, for which there is currently no specific treatme.
RP-A501 delivers a functional human LAMP2B transgene (AAVLAMP2B) from an AAV9 vect.
After a single intravenous injection, these genes will be stable and expressed in episomal form in non-dividing cells, thus effectively treating monogenic genetic diseas.
Data presented here include previously disclosed safety and efficacy data in young adult and adolescent patients at low dose (7 x 1013 GC/kg; n=3) and high dose (1 x 1014 GC/kg; n=2) , and safety data from a low-dose (7 x 1013 GC/kg; n=2) pediatric coho.
RP-A501 was well tolerated in low-dose cohorts of young adults and adolescen.
All young adult and adolescent patients showed disease changes in molecular, echocardiographic, and patient functional paramete.
In the pediatric cohort, RP-A501 was well tolerated in both patien.
The patient was observed to have a normal platelet range, reduced complement activation, and no complement-related adverse even.
The two patients received prophylactic treatment with a modified immunosuppressive regim.
To date, no apparent immediate or delayed toxicity has been observ.
➤ Lentivirus-mediated gene therapy RP-L301 for the treatment of pyruvate kinase deficiency Pyruvate kinase deficiency (PKD) is a rare monogenic red blood cell disorder caused by mutations in the PKLR gene encoding pyruvate kinase, which is a A key component of the erythrocyte glycolytic pathw.
Mutations in the PKLR gene lead to increased red blood cell destruction with varying degrees of anemia from mild to life-threateni.
RP-L301 is a gene therapy candidate drug for the treatment of PKD using lentivirus as a vect.
The data presented here are from two adult patients with severe anemia who were treated with RP-L30 At 18 months after infusion, both patients had sustained transgene expression, normalization of hemoglobin, improved hemolysis, no need for red blood cell transfusion after transplantation, and improved quality of li.
The safety profile of RP-L301 was favorable, with no IP-related serious adverse events 18 months after infusi.
Lentivirus-mediated gene therapy LV and γ-retrovirus belong to the same family of retroviruses, and both integrate their double-stranded DNA into the host genome, and then use the host's mechanism to transcribe their genes back into R.
Once replicated and returned to the cytoplasm, the viral RNA is translated and packaged into a new viral particle, which then buds out of the cell to complete the life cyc.
The infection efficiency of LV to dividing and non-dividing cells can reach 30%, while the infection efficiency of γ-retrovirus to most cells is <30% and depends on cell divisi.
Therefore, lentiviral vectors are currently the tool of choice for most hematopoietic stem cell (HSC) editi.
Although LV and γ-retrovirus have the same safety problem of possible insertional mutation, the latter will integrate into the 5' untranslated region of the gene, and the former will preferentially integrate into the coding region of the gene, thereby reducing the risk of carcinogenes.
Compared with recombinant adenovirus (Ad) and adeno-associated virus (AAV), LV can carry larger and more complex transgenes, and has the ability to integrate the transgene into the host genome for stable and long-term expression of the produ.
In addition to Rocket, many companies including Bluebird Bio, Orchard Therapeutics, Mustang Bio, Oxford BioMedica, AVROBIO, e.
are conducting research on lentiviral gene thera.
➤ Bluebird Bio Bluebird Bio is a biotechnology company focusing on the use of lentiviral gene therapy for the treatment of serious genetic diseas.
It has 3 main pipeline products, namely Beti-Cel (for the treatment of β-thalassemia), Lovo-Cel (for sickle cell disease) and Eli-Cel (for adrenoleukodystroph.
Beti-cel is Bluebird's first gene therapy and the world's first gene therapy product for T.
In early June 2019, Beti-cel was conditionally approved by the European Commission (EC) for the treatment of transfusion-dependent β-thalassemia patients 12 years of age and older with a non-β0/β0 genotype, however it was delayed by the EMA due to data and manufacturing issues It was not commercially available for the first time in Germany until January 2020, under the trade name Zynteg.
➤ Orchard Therapeutics Orchard Therapeutics' OTL-103 is an autologous hematopoietic stem cell gene therapy consisting of CD34+ cells transfected in vitro with a lentiviral vector encoding the Wiskott-Aldrich syndrome (WAS) gene, designed to treat Wiskott-Aldrich syndrome by a single injecti.
Aldrich syndro.
The therapy has been granted Orphan Drug Designation and Rare Pediatric Disease (RPD) designation by the F.
Currently, Phase 3 clinical trials evaluating efficacy and safety are underw.
OTL-101 is a hematopoietic stem and progenitor cell (HSPC) + patient's autologous CD34, CD34-positive HSCs were isolated from patients, and the correct adenosine deaminase (ADA) gene was transduced in vitro by self-inactivating lentivirus, using For the treatment of patients with severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCI.
Currently, OTL-101 has been granted orphan drug designation by the US FDA and EU EMA for the treatment of ADA-SC.
On May 11, 2021, OTL-101's ongoing Phase 1/2 clinical results demonstrated a 100% overall survival rate in ADA-SCID after 2 and 3 years of gene therapy studi.
OTL-200 is a lentiviral vector-based, autologous hematopoietic stem cell gene thera.
It uses a lentiviral vector containing human arylsulfatase A (ARSA) gene to transfect autologous differentiated CD34+ cells, so that the cells express aryl Sulfatase A, thereby treating metachromatic leukodystrophy (ML.
In November 2020, the US FDA approved the IND application for OTL-20
➤ Mustang Bio In November 2021, Mustang Bio announced that it had signed an exclusive license agreement with Leiden University Medical Center (LUMC) to obtain the first individual for the treatment of RAG1 severe combined immunodeficiency (RAG1-SCID) Extrinsic lentiviral gene thera.
The therapy, which involves low-dose conditioning prior to infusion of the patient's own genetically modified hematopoietic stem cells, is currently being evaluated in a phase 1/2 multicenter clinical trial in Euro.
An ongoing clinical trial recently enrolled its first patient, and more clinical sites are expected to be added in the near futu.
The RAG1-SCID project has been granted orphan drug designation by the European Medicines Agency (EM.
➤ Oxford BioMedica Oxford is a pioneer in lentiviral gene therapy and is the only supplier of lentiviral vectors for Novartis' CAR-T product Kymri.
In terms of product research and development, with the LentiVector® lentiviral vector delivery technology, the company has established good cooperative relations with first-tier pharmaceutical companies such as Sanofi and GSK, and has a huge source of funds, including process development and bioprocessing income and royalti.
In addition, the company's clinical research on gene therapy for ophthalmic diseases and Parkinson's disease is also progressing steadi.
➤ AVROBIO AVROBIO is working to advance several lentiviral gene therapies for lysosomal storage diseases including Fabry, Gaucher, cystinosis and Pompe disea.
The company designed a proprietary third-generation 4-plasmid lentiviral vector system, an efficient and proven gene transfer system for the stable addition of genes to a patient's own CD34+ cel.
AVROBIO's lentiviral approach is designed to enable treated stem cells to differentiate into many different cell types - including T cells, B cells and monocytes, each of which is expected to carry therapeutic gen.
When monocytes enter tissue they become macrophages and in the central nervous system (CNS) they become microgl.
Monocytes, macrophages and microglia carrying therapeutic genes are thought to have the ability to cross the blood-brain barrier and enter the C.
Once dispersed in the brain and central nervous system, these therapeutic cells may be able to slow, stop or even prevent disease progression in the central nervous syst.
➤ Bendao Gene On December 30, 2021, Shanghai Bendao Gene Technology .
, L.
announced to the public that the "Lentiviral Vector Transformation" jointly developed by the 920th Hospital of the Joint Logistics Support Force of the Chinese People's Liberation Army and Shanghai Bendao Gene Technology .
, L.
The clinical trial of "Safety and Effectiveness of Autologous CD34+ Hematopoietic Stem Cells in the Treatment of Transfusion-dependent β-Thalassemia" has achieved preliminary resul.
This is the first domestic report of a successful case of treating β-thalassemia based on lentiviral vector gene transduction technolo.
At present, Bendao Gene is actively promoting its IND application for β-thalassemia gene therapy based on lentiviral vect.
➤ Kanglin Bio-Kanglin Biotechnology (Hangzhou) .
, L.
has been focusing on the research and development of innovative gene therapy drugs since its establishment in 2015, targeting mostly diseases that seriously affect human health, such as AIDS, Parkinson's disease, hemophilia disease and thalassem.
Kanglin Bio is currently a rare company in the world with high-level R&D and production capabilities of lentivirus and adeno-associated virus vecto.
In May 2022, Kanglin Bio announced that its self-developed innovative gene therapy drug for thalassemia, KL003, has successfully cured 2 critically ill patients in a clinical trial initiated by the investigator, and its indicators are far better than those of the world's leading US bluebird creatu.
➤ Zhongji Zhiyao Zhongji Zhiyao's β-thalassemia gene therapy pipeline adopts gene replacement therapy, using lentivirus to introduce the correct β-globin gene into hematopoietic stem cells, and back to the patient to increase the expression of β-globin and promote the production of HbA , so as to achieve the purpose of treatment or cure for β-thalassem.
Among them, the in vitro hematopoietic stem cell erythroid differentiation platform was used to induce differentiation of CD34+ cells for 21 days, and the erythrocytes were collect.
At present, the pipeline has entered the stage of IIT clinical research and IND applicati.
Summary Several virus types have been investigated for use as viral vectors in gene therapy, including LV, AAV, and retrovirus.
Gene therapy using AAV as a delivery vehicle has made progress in a variety of indications, including a range of rare eye diseases, hemophilia, Huntington's disease, Fabry disease, glycogen storage diseases, and mo.
The strategy of most companies to develop gene therapy using lentivirus as a delivery vector is to transduce autologous stem cells with lentiviral vecto.
For many diseases for which hematopoietic stem cell transplantation is the only treatment method, lentiviral gene therapy has the potential to create new treatmen.
prospec.
However, lentiviruses also carry the risk of gene mutation due to random insertion, so there has been a concern that the integration of transgenes into certain sites in the genome may lead to the occurrence of canc.
For a long time in the future, viral vectors will still be the mainstream gene delivery too.
Faced with potential safety hazards, the improvement and optimization of viral vectors will be the top priority of researche.
References:https:// -at-the-25th-annual-meeting-of-the-american-society-of-gene-and-cell-therapy-asgct-/ Official website of each company - list of recent popular events - ▼ May 19 , Future-oriented CGT digital solutions ▼ On May 20, cryo-electron microscopy - high-tech new technologies break traditional thinking to help the development of new drugs▼ On May 22, 2022 TIL Cell Therapy Technology Innovation Forum
Positive data from pivotal Phase 2 clinical trials and Phase 1 clinical trials in Danon Disease (DD) and Pyruvate Kinase Deficiency (PK.
➤ Ex vivo lentivirus-mediated gene therapy RP-L102 in patients with Fanconi anemia Fanconi anemia (FA) is a rare pediatric disease, and approximately 60-70% of FA patients have mutations in the FANCA gene, which encodes A protein essential for DNA repa.
Mutated genes in FANCA cause chromosome breakage and increase susceptibility to oxidative and environmental stre.
Patients are prone to bone marrow dysfunction and cancer susceptibili.
RP-L102 is a gene therapy that uses lentiviral vector to deliver normal FANCA gene to transform hematopoietic stem cells in vit.
The data presented here are from an initial 9 of 12 patients who received RP-L10 Five of the nine evaluable patients showed increased resistance of bone marrow cells to the DNA-damaging drug MMC, indicating an enhanced ability of patients' bone marrow cells to repair DNA damage, meeting the trial's primary endpoi.
Peripheral VCN demonstrated that the sixth patient showed evidence of progressively increasing gene correcti.
Two other patients are currently only 12 months after treatment, and one patient developed progressive bone marrow failure and underwent a successful allogeneic transpla.
Three of the 12 treated patients had not yet reached the 12-month time poi.
The safety profile of RP-L102 was favorable, with no evidence of dysplasia, clonal dominance, or oncogenic integration; as previously reported, one patient experienced a grade 2 transient infusion-related reaction that subsequently resolv.
➤ AAV9-mediated gene therapy RP-A501 for the treatment of Danon's disease Danon's disease (DD) is a very rare and life-threatening disease due to the failure of basic biological processes in cells responsible for the removal and recycling of protei.
This is a rare X-linked autophagic vacuolar myopathy that causes multiple organ dysfunction, including the heart, skeletal muscle, nervous system, eye, and liver, for which there is currently no specific treatme.
RP-A501 delivers a functional human LAMP2B transgene (AAVLAMP2B) from an AAV9 vect.
After a single intravenous injection, these genes will be stable and expressed in episomal form in non-dividing cells, thus effectively treating monogenic genetic diseas.
Data presented here include previously disclosed safety and efficacy data in young adult and adolescent patients at low dose (7 x 1013 GC/kg; n=3) and high dose (1 x 1014 GC/kg; n=2) , and safety data from a low-dose (7 x 1013 GC/kg; n=2) pediatric coho.
RP-A501 was well tolerated in low-dose cohorts of young adults and adolescen.
All young adult and adolescent patients showed disease changes in molecular, echocardiographic, and patient functional paramete.
In the pediatric cohort, RP-A501 was well tolerated in both patien.
The patient was observed to have a normal platelet range, reduced complement activation, and no complement-related adverse even.
The two patients received prophylactic treatment with a modified immunosuppressive regim.
To date, no apparent immediate or delayed toxicity has been observ.
➤ Lentivirus-mediated gene therapy RP-L301 for the treatment of pyruvate kinase deficiency Pyruvate kinase deficiency (PKD) is a rare monogenic red blood cell disorder caused by mutations in the PKLR gene encoding pyruvate kinase, which is a A key component of the erythrocyte glycolytic pathw.
Mutations in the PKLR gene lead to increased red blood cell destruction with varying degrees of anemia from mild to life-threateni.
RP-L301 is a gene therapy candidate drug for the treatment of PKD using lentivirus as a vect.
The data presented here are from two adult patients with severe anemia who were treated with RP-L30 At 18 months after infusion, both patients had sustained transgene expression, normalization of hemoglobin, improved hemolysis, no need for red blood cell transfusion after transplantation, and improved quality of li.
The safety profile of RP-L301 was favorable, with no IP-related serious adverse events 18 months after infusi.
Lentivirus-mediated gene therapy LV and γ-retrovirus belong to the same family of retroviruses, and both integrate their double-stranded DNA into the host genome, and then use the host's mechanism to transcribe their genes back into R.
Once replicated and returned to the cytoplasm, the viral RNA is translated and packaged into a new viral particle, which then buds out of the cell to complete the life cyc.
The infection efficiency of LV to dividing and non-dividing cells can reach 30%, while the infection efficiency of γ-retrovirus to most cells is <30% and depends on cell divisi.
Therefore, lentiviral vectors are currently the tool of choice for most hematopoietic stem cell (HSC) editi.
Although LV and γ-retrovirus have the same safety problem of possible insertional mutation, the latter will integrate into the 5' untranslated region of the gene, and the former will preferentially integrate into the coding region of the gene, thereby reducing the risk of carcinogenes.
Compared with recombinant adenovirus (Ad) and adeno-associated virus (AAV), LV can carry larger and more complex transgenes, and has the ability to integrate the transgene into the host genome for stable and long-term expression of the produ.
In addition to Rocket, many companies including Bluebird Bio, Orchard Therapeutics, Mustang Bio, Oxford BioMedica, AVROBIO, e.
are conducting research on lentiviral gene thera.
➤ Bluebird Bio Bluebird Bio is a biotechnology company focusing on the use of lentiviral gene therapy for the treatment of serious genetic diseas.
It has 3 main pipeline products, namely Beti-Cel (for the treatment of β-thalassemia), Lovo-Cel (for sickle cell disease) and Eli-Cel (for adrenoleukodystroph.
Beti-cel is Bluebird's first gene therapy and the world's first gene therapy product for T.
In early June 2019, Beti-cel was conditionally approved by the European Commission (EC) for the treatment of transfusion-dependent β-thalassemia patients 12 years of age and older with a non-β0/β0 genotype, however it was delayed by the EMA due to data and manufacturing issues It was not commercially available for the first time in Germany until January 2020, under the trade name Zynteg.
➤ Orchard Therapeutics Orchard Therapeutics' OTL-103 is an autologous hematopoietic stem cell gene therapy consisting of CD34+ cells transfected in vitro with a lentiviral vector encoding the Wiskott-Aldrich syndrome (WAS) gene, designed to treat Wiskott-Aldrich syndrome by a single injecti.
Aldrich syndro.
The therapy has been granted Orphan Drug Designation and Rare Pediatric Disease (RPD) designation by the F.
Currently, Phase 3 clinical trials evaluating efficacy and safety are underw.
OTL-101 is a hematopoietic stem and progenitor cell (HSPC) + patient's autologous CD34, CD34-positive HSCs were isolated from patients, and the correct adenosine deaminase (ADA) gene was transduced in vitro by self-inactivating lentivirus, using For the treatment of patients with severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCI.
Currently, OTL-101 has been granted orphan drug designation by the US FDA and EU EMA for the treatment of ADA-SC.
On May 11, 2021, OTL-101's ongoing Phase 1/2 clinical results demonstrated a 100% overall survival rate in ADA-SCID after 2 and 3 years of gene therapy studi.
OTL-200 is a lentiviral vector-based, autologous hematopoietic stem cell gene thera.
It uses a lentiviral vector containing human arylsulfatase A (ARSA) gene to transfect autologous differentiated CD34+ cells, so that the cells express aryl Sulfatase A, thereby treating metachromatic leukodystrophy (ML.
In November 2020, the US FDA approved the IND application for OTL-20
➤ Mustang Bio In November 2021, Mustang Bio announced that it had signed an exclusive license agreement with Leiden University Medical Center (LUMC) to obtain the first individual for the treatment of RAG1 severe combined immunodeficiency (RAG1-SCID) Extrinsic lentiviral gene thera.
The therapy, which involves low-dose conditioning prior to infusion of the patient's own genetically modified hematopoietic stem cells, is currently being evaluated in a phase 1/2 multicenter clinical trial in Euro.
An ongoing clinical trial recently enrolled its first patient, and more clinical sites are expected to be added in the near futu.
The RAG1-SCID project has been granted orphan drug designation by the European Medicines Agency (EM.
➤ Oxford BioMedica Oxford is a pioneer in lentiviral gene therapy and is the only supplier of lentiviral vectors for Novartis' CAR-T product Kymri.
In terms of product research and development, with the LentiVector® lentiviral vector delivery technology, the company has established good cooperative relations with first-tier pharmaceutical companies such as Sanofi and GSK, and has a huge source of funds, including process development and bioprocessing income and royalti.
In addition, the company's clinical research on gene therapy for ophthalmic diseases and Parkinson's disease is also progressing steadi.
➤ AVROBIO AVROBIO is working to advance several lentiviral gene therapies for lysosomal storage diseases including Fabry, Gaucher, cystinosis and Pompe disea.
The company designed a proprietary third-generation 4-plasmid lentiviral vector system, an efficient and proven gene transfer system for the stable addition of genes to a patient's own CD34+ cel.
AVROBIO's lentiviral approach is designed to enable treated stem cells to differentiate into many different cell types - including T cells, B cells and monocytes, each of which is expected to carry therapeutic gen.
When monocytes enter tissue they become macrophages and in the central nervous system (CNS) they become microgl.
Monocytes, macrophages and microglia carrying therapeutic genes are thought to have the ability to cross the blood-brain barrier and enter the C.
Once dispersed in the brain and central nervous system, these therapeutic cells may be able to slow, stop or even prevent disease progression in the central nervous syst.
➤ Bendao Gene On December 30, 2021, Shanghai Bendao Gene Technology .
, L.
announced to the public that the "Lentiviral Vector Transformation" jointly developed by the 920th Hospital of the Joint Logistics Support Force of the Chinese People's Liberation Army and Shanghai Bendao Gene Technology .
, L.
The clinical trial of "Safety and Effectiveness of Autologous CD34+ Hematopoietic Stem Cells in the Treatment of Transfusion-dependent β-Thalassemia" has achieved preliminary resul.
This is the first domestic report of a successful case of treating β-thalassemia based on lentiviral vector gene transduction technolo.
At present, Bendao Gene is actively promoting its IND application for β-thalassemia gene therapy based on lentiviral vect.
➤ Kanglin Bio-Kanglin Biotechnology (Hangzhou) .
, L.
has been focusing on the research and development of innovative gene therapy drugs since its establishment in 2015, targeting mostly diseases that seriously affect human health, such as AIDS, Parkinson's disease, hemophilia disease and thalassem.
Kanglin Bio is currently a rare company in the world with high-level R&D and production capabilities of lentivirus and adeno-associated virus vecto.
In May 2022, Kanglin Bio announced that its self-developed innovative gene therapy drug for thalassemia, KL003, has successfully cured 2 critically ill patients in a clinical trial initiated by the investigator, and its indicators are far better than those of the world's leading US bluebird creatu.
➤ Zhongji Zhiyao Zhongji Zhiyao's β-thalassemia gene therapy pipeline adopts gene replacement therapy, using lentivirus to introduce the correct β-globin gene into hematopoietic stem cells, and back to the patient to increase the expression of β-globin and promote the production of HbA , so as to achieve the purpose of treatment or cure for β-thalassem.
Among them, the in vitro hematopoietic stem cell erythroid differentiation platform was used to induce differentiation of CD34+ cells for 21 days, and the erythrocytes were collect.
At present, the pipeline has entered the stage of IIT clinical research and IND applicati.
Summary Several virus types have been investigated for use as viral vectors in gene therapy, including LV, AAV, and retrovirus.
Gene therapy using AAV as a delivery vehicle has made progress in a variety of indications, including a range of rare eye diseases, hemophilia, Huntington's disease, Fabry disease, glycogen storage diseases, and mo.
The strategy of most companies to develop gene therapy using lentivirus as a delivery vector is to transduce autologous stem cells with lentiviral vecto.
For many diseases for which hematopoietic stem cell transplantation is the only treatment method, lentiviral gene therapy has the potential to create new treatmen.
prospec.
However, lentiviruses also carry the risk of gene mutation due to random insertion, so there has been a concern that the integration of transgenes into certain sites in the genome may lead to the occurrence of canc.
For a long time in the future, viral vectors will still be the mainstream gene delivery too.
Faced with potential safety hazards, the improvement and optimization of viral vectors will be the top priority of researche.
References:https:// -at-the-25th-annual-meeting-of-the-american-society-of-gene-and-cell-therapy-asgct-/ Official website of each company - list of recent popular events - ▼ May 19 , Future-oriented CGT digital solutions ▼ On May 20, cryo-electron microscopy - high-tech new technologies break traditional thinking to help the development of new drugs▼ On May 22, 2022 TIL Cell Therapy Technology Innovation Forum
