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CRISPR Revolution in Cell & Gene Therapy
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CRISPR has ushered in a new era for biomedicine, particularly in cell and gene therapies. These are exciting times for this field with clinical trials for sickle cell anemia and CAR T cancer therapies underway. Synthego’s recent virtual event- World CRISPR Day had sessions on all areas of genome engineering ranging from agriculture to stem cell research. In this article, we have summarized the key points from the Cell and Gene therapies session and included additional resources to learn further about this exciting field.
Dr. Waseem Qasim Speaks About Genome Edited Therapeutic T-Cells
Speaker Info:Dr. Qasim is a Clinician and Professor of Gene and Cell Therapy at the Institute of Child Health, University College London, and a consultant in paediatric immunology/BMT at Great Ormond Street Hospital, London. His research group combines clinical work in immunology & transplantation with gene therapy research, investigating the development and translational applications of genome editing tools like CRISPR-Cas9 and base editors.
Dr. Qasim is a Clinician and Professor of Gene and Cell Therapy at the Institute of Child Health, University College London, and a consultant in paediatric immunology/BMT at Great Ormond Street Hospital, London. His research group combines clinical work in immunology & transplantation with gene therapy research, investigating the development and translational applications of genome editing tools like CRISPR-Cas9 and base editors.
AbstractDr. Qasim’s presentation focused on CRISPR editing of CAR-T cells. He explained what engineered T-cells were and the key applications of CAR T-cell therapy. He also discussed the advantages and limitations of this method and elaborated on the need for bespoke products tailored for each individual patient. He then discussed a clinical trial involving the use of CRISPR-CAR19-edited allogeneic T cells in pediatric relapsed or refractory B-cell acute lymphoblastic leukemia. He hoped that this method would allow for treatment without any side effects.
Dr. Qasim’s presentation focused on CRISPR editing of CAR-T cells. He explained what engineered T-cells were and the key applications of CAR T-cell therapy. He also discussed the advantages and limitations of this method and elaborated on the need for bespoke products tailored for each individual patient. He then discussed a clinical trial involving the use of CRISPR-CAR19-edited allogeneic T cells in pediatric relapsed or refractory B-cell acute lymphoblastic leukemia. He hoped that this method would allow for treatment without any side effects.
Key takeaways
- Advantages of T- cells: Why they are ideal for genetic therapies: Dr. Qasim explained why T-cells were important and listed a number of reasons why they are ideal for genetic therapies. (watch on YouTube)
- Limitations of CAR strategies: Dr. Qasim briefly discussed some limitations of CAR strategies and explained how these could be overcome by genome editing. (watch on YouTube)
- Success of the first licensed product: YESCARTA is the first approved CAR-T cell therapy product to treat adults with certain types of non-Hodgkin lymphoma. These products are expensive because they are specifically manufactured for each patient using their own cells, but they have had high success rates and are changing the face of how we deal with some hard-to-treat leukemias. (watch on YouTube)
- TALEN and CRISPR clinical trials: The phase I clinical trials of UCART19 are now underway. Dr.Qasim’s lab is associated with a study that had recently entered a phase I clinical trial. The trial involves using CRISPR-CAR19-edited allogeneic T cells in pediatric relapsed or refractory B-cell acute lymphoblastic leukemia with the hope of disease treatment without any side effects. (watch on YouTube)
- Advantages of T- cells: Why they are ideal for genetic therapies: Dr. Qasim explained why T-cells were important and listed a number of reasons why they are ideal for genetic therapies. (watch on YouTube)
- Limitations of CAR strategies: Dr. Qasim briefly discussed some limitations of CAR strategies and explained how these could be overcome by genome editing. (watch on YouTube)
- Success of the first licensed product: YESCARTA is the first approved CAR-T cell therapy product to treat adults with certain types of non-Hodgkin lymphoma. These products are expensive because they are specifically manufactured for each patient using their own cells, but they have had high success rates and are changing the face of how we deal with some hard-to-treat leukemias. (watch on YouTube)
- TALEN and CRISPR clinical trials: The phase I clinical trials of UCART19 are now underway. Dr.Qasim’s lab is associated with a study that had recently entered a phase I clinical trial. The trial involves using CRISPR-CAR19-edited allogeneic T cells in pediatric relapsed or refractory B-cell acute lymphoblastic leukemia with the hope of disease treatment without any side effects. (watch on YouTube)
Dr. Donald Kohn On Gene Editing In Hematopoietic Stem Cells
Speaker Info:
AbstractDr. Kohn spoke about his area of research focus--using autologous hematopoietic stem cells to develop gene therapy methods to treat blood cell diseases, such as severe combined immune deficiency and sickle cell anemia. He also mentioned an upcoming phase I trial involving nine patients with severe sickle cell disease. The first patient will be enrolled in 2021 and will receive CRISPR-Cas9-corrected hematopoietic stem cells.
Dr. Kohn spoke about his area of research focus--using autologous hematopoietic stem cells to develop gene therapy methods to treat blood cell diseases, such as severe combined immune deficiency and sickle cell anemia. He also mentioned an upcoming phase I trial involving nine patients with severe sickle cell disease. The first patient will be enrolled in 2021 and will receive CRISPR-Cas9-corrected hematopoietic stem cells.
Key takeaways
- Repair of Sickle Cell Mutation in β-globin gene: Every single patient with the disease has the same base pair change, making sickle cell a very attractive target for editing. (watch on YouTube)
- In Vitro Erythroid Differentiation of HSC to RBC: The patients of the sickle cell disease only make hemoglobin S, but CRISPR editing of the HSCs, Dr. Kohn’s team was able to demonstrate that these patients were now able to produce hemoglobin A.While they were able to show 15-20% gene correction, there was also a high indel frequency at the target site. (watch on YouTube)
- Quest for Improved HDR / NHEJ in HSC: The results of the above mentioned studies, sent them on a quest to find ways of improving HDR / NHEJ outcomes in HSCs like using AAV6 for donor, use of Cas9 fusion proteins etc. (watch on YouTube)
- Analysis of edited CD34+ PBSC in NSG mice: Dr. Kohn discussed the results of the study by Romero et.al. that involved the comparison of activity of ZNFs and CRISPR-Cas9 for editing and homologous donor templates. (watch on YouTube)
- Comparison of Medium conditions on gene editing outcomes: Dr.Kohn’s team compared their standard medium conditions to those of De Ravin et al. (watch on YouTube)
- Curing Sickle Cell Disease using CRISPR-Cas9 editing: Their team developed a GMP- compliant manufacturing protocol that uses non-viral gene editing to correct SCD mutation. (watch on YouTube)
- Repair of Sickle Cell Mutation in β-globin gene: Every single patient with the disease has the same base pair change, making sickle cell a very attractive target for editing. (watch on YouTube)
- In Vitro Erythroid Differentiation of HSC to RBC: The patients of the sickle cell disease only make hemoglobin S, but CRISPR editing of the HSCs, Dr. Kohn’s team was able to demonstrate that these patients were now able to produce hemoglobin A.While they were able to show 15-20% gene correction, there was also a high indel frequency at the target site. (watch on YouTube)
- Quest for Improved HDR / NHEJ in HSC: The results of the above mentioned studies, sent them on a quest to find ways of improving HDR / NHEJ outcomes in HSCs like using AAV6 for donor, use of Cas9 fusion proteins etc. (watch on YouTube)
- Analysis of edited CD34+ PBSC in NSG mice: Dr. Kohn discussed the results of the study by Romero et.al. that involved the comparison of activity of ZNFs and CRISPR-Cas9 for editing and homologous donor templates. (watch on YouTube)
- Comparison of Medium conditions on gene editing outcomes: Dr.Kohn’s team compared their standard medium conditions to those of De Ravin et al. (watch on YouTube)
- Curing Sickle Cell Disease using CRISPR-Cas9 editing: Their team developed a GMP- compliant manufacturing protocol that uses non-viral gene editing to correct SCD mutation. (watch on YouTube)
Dr. Matthew Porteus On Translational Genome Editing For Sickle Cell Disease
Speaker Info:Dr. Porteus is an Associate Professor of Pediatrics, Divisions of Hematology/Oncology and Human Gene Therapy, at Stanford School of Medicine. He is also a member of Synthego’s advisory board and co-founder of CRISPR Therapeutics and Graphite Bio.
Dr. Porteus is an Associate Professor of Pediatrics, Divisions of Hematology/Oncology and Human Gene Therapy, at Stanford School of Medicine. He is also a member of Synthego’s advisory board and co-founder of CRISPR Therapeutics and Graphite Bio.
AbstractDr. Porteus spoke about the tremendous impact CRISPR has had on translational genome editing. He stressed the importance of cell and gene therapies using stem cells that have potential lifetime durability. His lab is focused on approaches relying on the homology-directed repair pathway to create small nucleotide changes for sickle cell disease treatment. Their recent methodology has shown promising results and has been filed to the FDA for clinical trial approval for ex vivo therapy.
Dr. Porteus spoke about the tremendous impact CRISPR has had on translational genome editing. He stressed the importance of cell and gene therapies using stem cells that have potential lifetime durability. His lab is focused on approaches relying on the homology-directed repair pathway to create small nucleotide changes for sickle cell disease treatment. Their recent methodology has shown promising results and has been filed to the FDA for clinical trial approval for ex vivo therapy.
Key takeaways
- History of Medicine: Dr. Porteus spoke about the history of medicine starting from sanitation and clean water to the current advances involving living drugs - Cell and gene medicine and microbiome manipulation. (watch on YouTube)
- Multiple uses of Homologous Recombination Pathway: Dr. Proteus explained what homologous recombination is and what it is used for. (watch on YouTube)
- Ex Vivo Gene Targeting System using RNP and a non-integrating virus: Dr. Porteus explained the ex vivo gene targeting system that was developed by his lab and how it works. He also spoke about how this system could be applied. (watch on YouTube)
- Applications of Stem Cell Engineering in Synthetic Biology: Dr. Porteus explained how stem cell engineering could be used in synthetic biology. He specifically focussed on the treatment for sickle cell disease using cell therapies. (watch on YouTube)
- Phase I/II Clinical trial for correction of Sickle Cell disease: Dr. Porteus spoke about the clinical trial that will be starting phase I/II in 2021. In this trial, the patient derived hematopoietic stem cells would be edited using nuclease modified homologous recombination and then the corrected cells would be transplanted back into the patient. (watch on YouTube)
- History of Medicine: Dr. Porteus spoke about the history of medicine starting from sanitation and clean water to the current advances involving living drugs - Cell and gene medicine and microbiome manipulation. (watch on YouTube)
- Multiple uses of Homologous Recombination Pathway: Dr. Proteus explained what homologous recombination is and what it is used for. (watch on YouTube)
- Ex Vivo Gene Targeting System using RNP and a non-integrating virus: Dr. Porteus explained the ex vivo gene targeting system that was developed by his lab and how it works. He also spoke about how this system could be applied. (watch on YouTube)
- Applications of Stem Cell Engineering in Synthetic Biology: Dr. Porteus explained how stem cell engineering could be used in synthetic biology. He specifically focussed on the treatment for sickle cell disease using cell therapies. (watch on YouTube)
- Phase I/II Clinical trial for correction of Sickle Cell disease: Dr. Porteus spoke about the clinical trial that will be starting phase I/II in 2021. In this trial, the patient derived hematopoietic stem cells would be edited using nuclease modified homologous recombination and then the corrected cells would be transplanted back into the patient. (watch on YouTube)
6 Conferences And Webinars On Cell And Gene Therapies To Watch Out For
Interested in learning more about cell and gene therapies? We’ve compiled a list of conferences and other virtual events for you. Check them out to stay in tune with the latest research in this field.
1. Cell And Gene Therapy 2021Date: 18th & 19th of May 2021
Location: Gothenburg, Sweden
Description: This interactive 2-day conference by ELRIG will host world leading scientists from academia and industry to discuss current and future perspectives of Cell and Gene Therapies.
Date: 18th & 19th of May 2021
Location: Gothenburg, Sweden
Description: This interactive 2-day conference by ELRIG will host world leading scientists from academia and industry to discuss current and future perspectives of Cell and Gene Therapies.
2. Cell And Gene Therapy DayDate: 23rd February, 2021
Location: Virtual Event
Description: Cell & Gene Therapy Day convenes stakeholders from the business and science communities to report on the latest progress in the field to fight a wider range of cancers beyond hematologic malignancies.
Date: 23rd February, 2021
Location: Virtual Event
Description: Cell & Gene Therapy Day convenes stakeholders from the business and science communities to report on the latest progress in the field to fight a wider range of cancers beyond hematologic malignancies.
3. American Society of Gene and Cell Therapy- 24th Annual MeetingDate: 12-15th of May, 2021
Location: Oregon Convention Center, Portland, Oregon
Description: The Annual Meeting provides an international forum where the latest advancements in gene and cell therapy are presented and discussed. ASGCT's annual meeting has over 3,400 professionals including scientists, physicians, and patient advocates.
Date: 12-15th of May, 2021
Location: Oregon Convention Center, Portland, Oregon
Description: The Annual Meeting provides an international forum where the latest advancements in gene and cell therapy are presented and discussed. ASGCT's annual meeting has over 3,400 professionals including scientists, physicians, and patient advocates.
4. International Conference on Cell and Gene TherapyDate: 19-21st July, 2021
Location: Paris, France
Description: This conference aims to bring together leading scientists, academic researchers and scholars to exchange and share their research on all aspects of Cell and Gene Therapy.
Date: 19-21st July, 2021
Location: Paris, France
Description: This conference aims to bring together leading scientists, academic researchers and scholars to exchange and share their research on all aspects of Cell and Gene Therapy.
5. 11th International Conference and Exhibition on Advanced Cell and Gene TherapyDate: 15-16 March 2021
Location: Virtual event - Webinar
Description: This Cell and Gene Therapy Conference aims to ameliorate the knowledge, awareness, and education on Cell and Gene Therapy leading to the discovery of Genetic and Cellular Therapies, which aid to alleviate human disease.
Date: 15-16 March 2021
Location: Virtual event - Webinar
Description: This Cell and Gene Therapy Conference aims to ameliorate the knowledge, awareness, and education on Cell and Gene Therapy leading to the discovery of Genetic and Cellular Therapies, which aid to alleviate human disease.
6. 7th Annual Cell & Gene Therapy Congress 2021Date: Oct 2021 (tentative)
Location: Novotel London West, United Kingdom
Description: The 7th Annual Cell & Gene Therapy Congress 2021 brings together senior attendees from pharmaceutical, biopharmaceutical, biotechnology, diagnostics, CRO and solution provider companies, academic and government institutions.
Date: Oct 2021 (tentative)
Location: Novotel London West, United Kingdom
Description: The 7th Annual Cell & Gene Therapy Congress 2021 brings together senior attendees from pharmaceutical, biopharmaceutical, biotechnology, diagnostics, CRO and solution provider companies, academic and government institutions.
Best Resources To Learn More About Cell And Gene Therapy
Interested in learning more about cell and gene therapies? Whether you are a beginner looking to gain some knowledge or an advanced researcher in the field, we’ve got you covered. Check out the list of resources we’ve handpicked for every knowledge level.
1. CRISPR is Ushering in a New Era in Cell and Gene TherapiesType: Cell and Gene Therapy workflow
Knowledge Level: Advanced
Description: Synthego’s high quality, synthetic sgRNA seamlessly moves your cell and gene therapy research from discovery to the clinic. This article walks you through every step in the workflow.
Type: Cell and Gene Therapy workflow
Knowledge Level: Advanced
Description: Synthego’s high quality, synthetic sgRNA seamlessly moves your cell and gene therapy research from discovery to the clinic. This article walks you through every step in the workflow.
2. PublicationsType: Publications
Knowledge Level: Intermediate
Description: This page lists over 60 publications in cell and gene therapy that specifically used CRISPR technology.
Type: Publications
Knowledge Level: Intermediate
Description: This page lists over 60 publications in cell and gene therapy that specifically used CRISPR technology.
3. CRISPR to the Clinic and BeyondType: Webinar
Knowledge Level: Intermediate
Description: Learn about clinical applications of CRISPR and how Synthego can support you from early-phase research, through process development, and into the clinic, highlighting our new sgRNA GMP manufacturing capabilities.
Type: Webinar
Knowledge Level: Intermediate
Description: Learn about clinical applications of CRISPR and how Synthego can support you from early-phase research, through process development, and into the clinic, highlighting our new sgRNA GMP manufacturing capabilities.
4. Cell And GeneType: Website
Knowledge level: All levels
Description: This website covers all topics related to cell & gene therapies.
Type: Website
Knowledge level: All levels
Description: This website covers all topics related to cell & gene therapies.
5. Cell & Gene Therapy Learning CenterType: Learning Center
Knowledge Level: All levels
Description: This learning center is a great resource to learn about topics ranging from fundamentals of cell and gene therapy to advanced research in the field.
Type: Learning Center
Knowledge Level: All levels
Description: This learning center is a great resource to learn about topics ranging from fundamentals of cell and gene therapy to advanced research in the field.
6. Approved Cellular and Gene Therapy ProductsType: Products
Knowledge Level: Advanced
Description: If you are curious about the approved cell & therapy products, check out this list on the FDA website.
We hope you enjoyed reading this article. If you are interested in learning more about CRISPR, be sure to follow CRISPR in the news. You can also keep up with the latest CRISPR news by subscribing to our blog, or by following us on Twitter or Facebook!
Type: Products
Knowledge Level: Advanced
Description: If you are curious about the approved cell & therapy products, check out this list on the FDA website.
We hope you enjoyed reading this article. If you are interested in learning more about CRISPR, be sure to follow CRISPR in the news. You can also keep up with the latest CRISPR news by subscribing to our blog, or by following us on Twitter or Facebook!
