SlideShare a Scribd company logo
Biologic and
Clinical Efficacy
of LentiGlobin
for Sickle Cell
Disease
Journal Article on
Recent Advances in
Genetics
BEST FOR You
O R G A N I C S C O M P A N Y
Background
Sickle cell disease is characterized
by the painful recurrence of vaso-
occlusive events. Gene therapy with
the use of LentiGlobin for sickle cell
disease (bb1111:lovotibeglolene
autotemcel)consist of autologous
transplantation of hematopoietic
stem and progenitor cells
transduced with the BB305 lentiviral
vector encoding a modified B-globin
gene, which produces an
antisickling hemoglobin, HbAT87Q
2
BEST FOR You
O R G A N I C S C O M P A N Y
3
Sickle Cell
Single point mutation in the gene
encoding B-globin (HBB)
Patients with sickle cell disease may suffer
Vas0-occlusive events
Progressive vasculopathy
Chronic Hemolytic Anemia
BEST FOR You
O R G A N I C S C O M P A N Y
4
Treatment for Sickle Cell Anemia
 HLA-matched sibling allogenic
hematopoietic treatment
BEST FOR You
O R G A N I C S C O M P A N Y
5
LENTIGLOBIN
 A gene therapy consist of the
autologous transplantation of
hematopoietic stem and progenitor
cells (HSPCs) transduced with the
BB305 lentiviral vector coding for a
modified B-globin gene
BEST FOR You
O R G A N I C S C O M P A N Y
6
 As the modified B-globin gene is
produced, it resulted to the production
of an antisickling hemoglobin, HbAT87Q
 HbAT87Q is a modified adult hemoglobin
with an amino acid substitution
(threonine to glutamine at position 87)
BEST FOR You
O R G A N I C S C O M P A N Y
7
BEST FOR You
O R G A N I C S C O M P A N Y
8
TRIAL DESIGN AND OVERSIGHT
• This is nonrandomized and single-dose
cilinical trial conducted at 11 sites across
United States in collaboration with the
Bluebird Bio
BEST FOR You
O R G A N I C S C O M P A N Y
9
ENTRY CRITERIA AND PATIENT
POPULATION
• Eligible patient were between 12 to 50 years
of age, had received diagnosis of sickle cell
disease
• 24 month history of active treatment of
sickle cell disease
• No opportunity for matched HLA-identical
hematopoietic-cell donation.
BEST FOR You
O R G A N I C S C O M P A N Y
10
TRIAL PROCEDURES
• Precollection preparation
• Collection through plerixafor mobilization
and apheresis
• Refined LentiGlobin manufacturing process
through the ex vivo transduction of HSPC
with the BB305 letiviral vector
BEST FOR You
O R G A N I C S C O M P A N Y
11
BEST FOR You
O R G A N I C S C O M P A N Y
12
BEST FOR You
O R G A N I C S C O M P A N Y
13
Conclusion
 Sickle cell disease is a lifelong, genetic disorder and the effect
of one-time gene therapy requires a long time evaluation.
 It remains to be determined whether the effects of
LentiGlobin gene therapy are lifelong, as anticipated on the
basis of durability in clinical and precilinical studies and in
studies of allogenic hematopoietic stem cell transplantation.

More Related Content

Similar to Biologic and Clinical Efficacy of LentiGlobin for Sickle.pptx

Assignment bb group c hdn
Assignment bb group c   hdnAssignment bb group c   hdn
Assignment bb group c hdn
Rifa Razemin
 
br-54-149.pdf
br-54-149.pdfbr-54-149.pdf
br-54-149.pdf
ElsianaLaurencia1
 
Sickle Cell Disease
Sickle Cell DiseaseSickle Cell Disease
Sickle Cell Disease
najmaldin saki
 
Clonal b cells in patients with hepatitis c virus–associated mixed
Clonal b cells in patients with hepatitis c virus–associated mixedClonal b cells in patients with hepatitis c virus–associated mixed
Clonal b cells in patients with hepatitis c virus–associated mixed
Clinical Immunology Laboratory, HMRUO, Oran.
 
Sickle cell disease
Sickle cell diseaseSickle cell disease
Sickle cell disease
Simon Mwasambungu
 
Blood Transfusion Center by Slidesgo.pptx
Blood Transfusion Center by Slidesgo.pptxBlood Transfusion Center by Slidesgo.pptx
Blood Transfusion Center by Slidesgo.pptx
SAAAE
 
clinical.pptx
clinical.pptxclinical.pptx
clinical.pptx
Marwa Khalifa
 
Hemoglobinopathies - Oct 2014
Hemoglobinopathies - Oct 2014Hemoglobinopathies - Oct 2014
Hemoglobinopathies - Oct 2014
derosaMSKCC
 
Sickle cell disease.ppt
Sickle cell disease.pptSickle cell disease.ppt
Sickle cell disease.ppt
AbdulKaderSouid
 
Abo incompatibility safiullah
Abo incompatibility safiullahAbo incompatibility safiullah
Abo incompatibility safiullah
Safiullah Sulaimankhil
 
Blood component therapy
Blood component therapy  Blood component therapy
Blood component therapy
Rakesh Verma
 
Chronic HEP B
Chronic HEP BChronic HEP B
Chronic HEP B
gattupruthvi
 
Gal-9 Sigma Xi ppt
Gal-9 Sigma Xi pptGal-9 Sigma Xi ppt
Gal-9 Sigma Xi ppt
LeeSengLau1
 
Akram et al-2018-journal_of_viral_hepatitis
Akram et al-2018-journal_of_viral_hepatitisAkram et al-2018-journal_of_viral_hepatitis
Akram et al-2018-journal_of_viral_hepatitis
Dr.Arifa Akram
 
Heme conf 10 08-2015 - dr cho
Heme conf 10 08-2015 - dr  choHeme conf 10 08-2015 - dr  cho
Heme conf 10 08-2015 - dr cho
derosaMSKCC
 
Verhovsek_et_al-2012-Pediatric_Blood_&_Cancer
Verhovsek_et_al-2012-Pediatric_Blood_&_CancerVerhovsek_et_al-2012-Pediatric_Blood_&_Cancer
Verhovsek_et_al-2012-Pediatric_Blood_&_Cancer
Ibi Wilcox
 
Clinical Case Study
Clinical Case StudyClinical Case Study
Clinical Case Study
Ashley Hamilton
 
Granulocyte antigens & antibodies and their role in transfusion medicinepptx
Granulocyte antigens & antibodies and their role in transfusion medicinepptxGranulocyte antigens & antibodies and their role in transfusion medicinepptx
Granulocyte antigens & antibodies and their role in transfusion medicinepptx
DrShinyKajal
 
RBC TF.pptx
RBC TF.pptxRBC TF.pptx
RBC TF.pptx
TanitavanNiekerk
 
blood bank seminar
blood bank seminarblood bank seminar
blood bank seminar
ala'a abdallah
 

Similar to Biologic and Clinical Efficacy of LentiGlobin for Sickle.pptx (20)

Assignment bb group c hdn
Assignment bb group c   hdnAssignment bb group c   hdn
Assignment bb group c hdn
 
br-54-149.pdf
br-54-149.pdfbr-54-149.pdf
br-54-149.pdf
 
Sickle Cell Disease
Sickle Cell DiseaseSickle Cell Disease
Sickle Cell Disease
 
Clonal b cells in patients with hepatitis c virus–associated mixed
Clonal b cells in patients with hepatitis c virus–associated mixedClonal b cells in patients with hepatitis c virus–associated mixed
Clonal b cells in patients with hepatitis c virus–associated mixed
 
Sickle cell disease
Sickle cell diseaseSickle cell disease
Sickle cell disease
 
Blood Transfusion Center by Slidesgo.pptx
Blood Transfusion Center by Slidesgo.pptxBlood Transfusion Center by Slidesgo.pptx
Blood Transfusion Center by Slidesgo.pptx
 
clinical.pptx
clinical.pptxclinical.pptx
clinical.pptx
 
Hemoglobinopathies - Oct 2014
Hemoglobinopathies - Oct 2014Hemoglobinopathies - Oct 2014
Hemoglobinopathies - Oct 2014
 
Sickle cell disease.ppt
Sickle cell disease.pptSickle cell disease.ppt
Sickle cell disease.ppt
 
Abo incompatibility safiullah
Abo incompatibility safiullahAbo incompatibility safiullah
Abo incompatibility safiullah
 
Blood component therapy
Blood component therapy  Blood component therapy
Blood component therapy
 
Chronic HEP B
Chronic HEP BChronic HEP B
Chronic HEP B
 
Gal-9 Sigma Xi ppt
Gal-9 Sigma Xi pptGal-9 Sigma Xi ppt
Gal-9 Sigma Xi ppt
 
Akram et al-2018-journal_of_viral_hepatitis
Akram et al-2018-journal_of_viral_hepatitisAkram et al-2018-journal_of_viral_hepatitis
Akram et al-2018-journal_of_viral_hepatitis
 
Heme conf 10 08-2015 - dr cho
Heme conf 10 08-2015 - dr  choHeme conf 10 08-2015 - dr  cho
Heme conf 10 08-2015 - dr cho
 
Verhovsek_et_al-2012-Pediatric_Blood_&_Cancer
Verhovsek_et_al-2012-Pediatric_Blood_&_CancerVerhovsek_et_al-2012-Pediatric_Blood_&_Cancer
Verhovsek_et_al-2012-Pediatric_Blood_&_Cancer
 
Clinical Case Study
Clinical Case StudyClinical Case Study
Clinical Case Study
 
Granulocyte antigens & antibodies and their role in transfusion medicinepptx
Granulocyte antigens & antibodies and their role in transfusion medicinepptxGranulocyte antigens & antibodies and their role in transfusion medicinepptx
Granulocyte antigens & antibodies and their role in transfusion medicinepptx
 
RBC TF.pptx
RBC TF.pptxRBC TF.pptx
RBC TF.pptx
 
blood bank seminar
blood bank seminarblood bank seminar
blood bank seminar
 

Recently uploaded

bordetella pertussis.................................ppt
bordetella pertussis.................................pptbordetella pertussis.................................ppt
bordetella pertussis.................................ppt
kejapriya1
 
原版制作(carleton毕业证书)卡尔顿大学毕业证硕士文凭原版一模一样
原版制作(carleton毕业证书)卡尔顿大学毕业证硕士文凭原版一模一样原版制作(carleton毕业证书)卡尔顿大学毕业证硕士文凭原版一模一样
原版制作(carleton毕业证书)卡尔顿大学毕业证硕士文凭原版一模一样
yqqaatn0
 
8.Isolation of pure cultures and preservation of cultures.pdf
8.Isolation of pure cultures and preservation of cultures.pdf8.Isolation of pure cultures and preservation of cultures.pdf
8.Isolation of pure cultures and preservation of cultures.pdf
by6843629
 
molar-distalization in orthodontics-seminar.pptx
molar-distalization in orthodontics-seminar.pptxmolar-distalization in orthodontics-seminar.pptx
molar-distalization in orthodontics-seminar.pptx
Anagha Prasad
 
在线办理(salfor毕业证书)索尔福德大学毕业证毕业完成信一模一样
在线办理(salfor毕业证书)索尔福德大学毕业证毕业完成信一模一样在线办理(salfor毕业证书)索尔福德大学毕业证毕业完成信一模一样
在线办理(salfor毕业证书)索尔福德大学毕业证毕业完成信一模一样
vluwdy49
 
Compexometric titration/Chelatorphy titration/chelating titration
Compexometric titration/Chelatorphy titration/chelating titrationCompexometric titration/Chelatorphy titration/chelating titration
Compexometric titration/Chelatorphy titration/chelating titration
Vandana Devesh Sharma
 
Unlocking the mysteries of reproduction: Exploring fecundity and gonadosomati...
Unlocking the mysteries of reproduction: Exploring fecundity and gonadosomati...Unlocking the mysteries of reproduction: Exploring fecundity and gonadosomati...
Unlocking the mysteries of reproduction: Exploring fecundity and gonadosomati...
AbdullaAlAsif1
 
THEMATIC APPERCEPTION TEST(TAT) cognitive abilities, creativity, and critic...
THEMATIC  APPERCEPTION  TEST(TAT) cognitive abilities, creativity, and critic...THEMATIC  APPERCEPTION  TEST(TAT) cognitive abilities, creativity, and critic...
THEMATIC APPERCEPTION TEST(TAT) cognitive abilities, creativity, and critic...
Abdul Wali Khan University Mardan,kP,Pakistan
 
Immersive Learning That Works: Research Grounding and Paths Forward
Immersive Learning That Works: Research Grounding and Paths ForwardImmersive Learning That Works: Research Grounding and Paths Forward
Immersive Learning That Works: Research Grounding and Paths Forward
Leonel Morgado
 
Authoring a personal GPT for your research and practice: How we created the Q...
Authoring a personal GPT for your research and practice: How we created the Q...Authoring a personal GPT for your research and practice: How we created the Q...
Authoring a personal GPT for your research and practice: How we created the Q...
Leonel Morgado
 
Basics of crystallography, crystal systems, classes and different forms
Basics of crystallography, crystal systems, classes and different formsBasics of crystallography, crystal systems, classes and different forms
Basics of crystallography, crystal systems, classes and different forms
MaheshaNanjegowda
 
Eukaryotic Transcription Presentation.pptx
Eukaryotic Transcription Presentation.pptxEukaryotic Transcription Presentation.pptx
Eukaryotic Transcription Presentation.pptx
RitabrataSarkar3
 
Micronuclei test.M.sc.zoology.fisheries.
Micronuclei test.M.sc.zoology.fisheries.Micronuclei test.M.sc.zoology.fisheries.
Micronuclei test.M.sc.zoology.fisheries.
Aditi Bajpai
 
Remote Sensing and Computational, Evolutionary, Supercomputing, and Intellige...
Remote Sensing and Computational, Evolutionary, Supercomputing, and Intellige...Remote Sensing and Computational, Evolutionary, Supercomputing, and Intellige...
Remote Sensing and Computational, Evolutionary, Supercomputing, and Intellige...
University of Maribor
 
Thornton ESPP slides UK WW Network 4_6_24.pdf
Thornton ESPP slides UK WW Network 4_6_24.pdfThornton ESPP slides UK WW Network 4_6_24.pdf
Thornton ESPP slides UK WW Network 4_6_24.pdf
European Sustainable Phosphorus Platform
 
Oedema_types_causes_pathophysiology.pptx
Oedema_types_causes_pathophysiology.pptxOedema_types_causes_pathophysiology.pptx
Oedema_types_causes_pathophysiology.pptx
muralinath2
 
mô tả các thí nghiệm về đánh giá tác động dòng khí hóa sau đốt
mô tả các thí nghiệm về đánh giá tác động dòng khí hóa sau đốtmô tả các thí nghiệm về đánh giá tác động dòng khí hóa sau đốt
mô tả các thí nghiệm về đánh giá tác động dòng khí hóa sau đốt
HongcNguyn6
 
Shallowest Oil Discovery of Turkiye.pptx
Shallowest Oil Discovery of Turkiye.pptxShallowest Oil Discovery of Turkiye.pptx
Shallowest Oil Discovery of Turkiye.pptx
Gokturk Mehmet Dilci
 
Describing and Interpreting an Immersive Learning Case with the Immersion Cub...
Describing and Interpreting an Immersive Learning Case with the Immersion Cub...Describing and Interpreting an Immersive Learning Case with the Immersion Cub...
Describing and Interpreting an Immersive Learning Case with the Immersion Cub...
Leonel Morgado
 
The debris of the ‘last major merger’ is dynamically young
The debris of the ‘last major merger’ is dynamically youngThe debris of the ‘last major merger’ is dynamically young
The debris of the ‘last major merger’ is dynamically young
Sérgio Sacani
 

Recently uploaded (20)

bordetella pertussis.................................ppt
bordetella pertussis.................................pptbordetella pertussis.................................ppt
bordetella pertussis.................................ppt
 
原版制作(carleton毕业证书)卡尔顿大学毕业证硕士文凭原版一模一样
原版制作(carleton毕业证书)卡尔顿大学毕业证硕士文凭原版一模一样原版制作(carleton毕业证书)卡尔顿大学毕业证硕士文凭原版一模一样
原版制作(carleton毕业证书)卡尔顿大学毕业证硕士文凭原版一模一样
 
8.Isolation of pure cultures and preservation of cultures.pdf
8.Isolation of pure cultures and preservation of cultures.pdf8.Isolation of pure cultures and preservation of cultures.pdf
8.Isolation of pure cultures and preservation of cultures.pdf
 
molar-distalization in orthodontics-seminar.pptx
molar-distalization in orthodontics-seminar.pptxmolar-distalization in orthodontics-seminar.pptx
molar-distalization in orthodontics-seminar.pptx
 
在线办理(salfor毕业证书)索尔福德大学毕业证毕业完成信一模一样
在线办理(salfor毕业证书)索尔福德大学毕业证毕业完成信一模一样在线办理(salfor毕业证书)索尔福德大学毕业证毕业完成信一模一样
在线办理(salfor毕业证书)索尔福德大学毕业证毕业完成信一模一样
 
Compexometric titration/Chelatorphy titration/chelating titration
Compexometric titration/Chelatorphy titration/chelating titrationCompexometric titration/Chelatorphy titration/chelating titration
Compexometric titration/Chelatorphy titration/chelating titration
 
Unlocking the mysteries of reproduction: Exploring fecundity and gonadosomati...
Unlocking the mysteries of reproduction: Exploring fecundity and gonadosomati...Unlocking the mysteries of reproduction: Exploring fecundity and gonadosomati...
Unlocking the mysteries of reproduction: Exploring fecundity and gonadosomati...
 
THEMATIC APPERCEPTION TEST(TAT) cognitive abilities, creativity, and critic...
THEMATIC  APPERCEPTION  TEST(TAT) cognitive abilities, creativity, and critic...THEMATIC  APPERCEPTION  TEST(TAT) cognitive abilities, creativity, and critic...
THEMATIC APPERCEPTION TEST(TAT) cognitive abilities, creativity, and critic...
 
Immersive Learning That Works: Research Grounding and Paths Forward
Immersive Learning That Works: Research Grounding and Paths ForwardImmersive Learning That Works: Research Grounding and Paths Forward
Immersive Learning That Works: Research Grounding and Paths Forward
 
Authoring a personal GPT for your research and practice: How we created the Q...
Authoring a personal GPT for your research and practice: How we created the Q...Authoring a personal GPT for your research and practice: How we created the Q...
Authoring a personal GPT for your research and practice: How we created the Q...
 
Basics of crystallography, crystal systems, classes and different forms
Basics of crystallography, crystal systems, classes and different formsBasics of crystallography, crystal systems, classes and different forms
Basics of crystallography, crystal systems, classes and different forms
 
Eukaryotic Transcription Presentation.pptx
Eukaryotic Transcription Presentation.pptxEukaryotic Transcription Presentation.pptx
Eukaryotic Transcription Presentation.pptx
 
Micronuclei test.M.sc.zoology.fisheries.
Micronuclei test.M.sc.zoology.fisheries.Micronuclei test.M.sc.zoology.fisheries.
Micronuclei test.M.sc.zoology.fisheries.
 
Remote Sensing and Computational, Evolutionary, Supercomputing, and Intellige...
Remote Sensing and Computational, Evolutionary, Supercomputing, and Intellige...Remote Sensing and Computational, Evolutionary, Supercomputing, and Intellige...
Remote Sensing and Computational, Evolutionary, Supercomputing, and Intellige...
 
Thornton ESPP slides UK WW Network 4_6_24.pdf
Thornton ESPP slides UK WW Network 4_6_24.pdfThornton ESPP slides UK WW Network 4_6_24.pdf
Thornton ESPP slides UK WW Network 4_6_24.pdf
 
Oedema_types_causes_pathophysiology.pptx
Oedema_types_causes_pathophysiology.pptxOedema_types_causes_pathophysiology.pptx
Oedema_types_causes_pathophysiology.pptx
 
mô tả các thí nghiệm về đánh giá tác động dòng khí hóa sau đốt
mô tả các thí nghiệm về đánh giá tác động dòng khí hóa sau đốtmô tả các thí nghiệm về đánh giá tác động dòng khí hóa sau đốt
mô tả các thí nghiệm về đánh giá tác động dòng khí hóa sau đốt
 
Shallowest Oil Discovery of Turkiye.pptx
Shallowest Oil Discovery of Turkiye.pptxShallowest Oil Discovery of Turkiye.pptx
Shallowest Oil Discovery of Turkiye.pptx
 
Describing and Interpreting an Immersive Learning Case with the Immersion Cub...
Describing and Interpreting an Immersive Learning Case with the Immersion Cub...Describing and Interpreting an Immersive Learning Case with the Immersion Cub...
Describing and Interpreting an Immersive Learning Case with the Immersion Cub...
 
The debris of the ‘last major merger’ is dynamically young
The debris of the ‘last major merger’ is dynamically youngThe debris of the ‘last major merger’ is dynamically young
The debris of the ‘last major merger’ is dynamically young
 

Biologic and Clinical Efficacy of LentiGlobin for Sickle.pptx

  • 1. Biologic and Clinical Efficacy of LentiGlobin for Sickle Cell Disease Journal Article on Recent Advances in Genetics
  • 2. BEST FOR You O R G A N I C S C O M P A N Y Background Sickle cell disease is characterized by the painful recurrence of vaso- occlusive events. Gene therapy with the use of LentiGlobin for sickle cell disease (bb1111:lovotibeglolene autotemcel)consist of autologous transplantation of hematopoietic stem and progenitor cells transduced with the BB305 lentiviral vector encoding a modified B-globin gene, which produces an antisickling hemoglobin, HbAT87Q 2
  • 3. BEST FOR You O R G A N I C S C O M P A N Y 3 Sickle Cell Single point mutation in the gene encoding B-globin (HBB) Patients with sickle cell disease may suffer Vas0-occlusive events Progressive vasculopathy Chronic Hemolytic Anemia
  • 4. BEST FOR You O R G A N I C S C O M P A N Y 4 Treatment for Sickle Cell Anemia  HLA-matched sibling allogenic hematopoietic treatment
  • 5. BEST FOR You O R G A N I C S C O M P A N Y 5 LENTIGLOBIN  A gene therapy consist of the autologous transplantation of hematopoietic stem and progenitor cells (HSPCs) transduced with the BB305 lentiviral vector coding for a modified B-globin gene
  • 6. BEST FOR You O R G A N I C S C O M P A N Y 6  As the modified B-globin gene is produced, it resulted to the production of an antisickling hemoglobin, HbAT87Q  HbAT87Q is a modified adult hemoglobin with an amino acid substitution (threonine to glutamine at position 87)
  • 7. BEST FOR You O R G A N I C S C O M P A N Y 7
  • 8. BEST FOR You O R G A N I C S C O M P A N Y 8 TRIAL DESIGN AND OVERSIGHT • This is nonrandomized and single-dose cilinical trial conducted at 11 sites across United States in collaboration with the Bluebird Bio
  • 9. BEST FOR You O R G A N I C S C O M P A N Y 9 ENTRY CRITERIA AND PATIENT POPULATION • Eligible patient were between 12 to 50 years of age, had received diagnosis of sickle cell disease • 24 month history of active treatment of sickle cell disease • No opportunity for matched HLA-identical hematopoietic-cell donation.
  • 10. BEST FOR You O R G A N I C S C O M P A N Y 10 TRIAL PROCEDURES • Precollection preparation • Collection through plerixafor mobilization and apheresis • Refined LentiGlobin manufacturing process through the ex vivo transduction of HSPC with the BB305 letiviral vector
  • 11. BEST FOR You O R G A N I C S C O M P A N Y 11
  • 12. BEST FOR You O R G A N I C S C O M P A N Y 12
  • 13. BEST FOR You O R G A N I C S C O M P A N Y 13 Conclusion  Sickle cell disease is a lifelong, genetic disorder and the effect of one-time gene therapy requires a long time evaluation.  It remains to be determined whether the effects of LentiGlobin gene therapy are lifelong, as anticipated on the basis of durability in clinical and precilinical studies and in studies of allogenic hematopoietic stem cell transplantation.