Cell Therapy News Volume 21.35 | Oct 5 2020

May 12, 2022

CTN 21.35 | Oct 5 2020

Cell Therapy News by STEMCELL Technologies

Vol. 21.35 – 5 October, 2020

TOP STORY

Bispecific
Anti-CD20, Anti-CD19 CAR T Cells for Relapsed B Cell Malignancies: A Phase I Dose Escalation and Expansion Trial

Researchers conducted a first-in-human trial of bispecific anti-CD20, anti-CD19 CAR T cells for relapsed, refractory B cell malignancies.
[Nature Medicine]

Abstract

Learn about retinal cell therapy using hESCs by watching Dr. Peter Coffey's webinar from the Nature Research Round Table on hPSC Quality

PUBLICATIONSRanked by the impact factor of the journal

Characteristics
of Anti-CD19 CAR T Cell Infusion Products Associated with Efficacy and Toxicity in Patients with Large B Cell Lymphomas

The authors performed single-cell RNA sequencing with capture-based cell identification on autologous axicabtagene ciloleucel anti-CD19 CAR T cell infusion products to identify transcriptomic features associated with efficacy and toxicity in 24 patients with large B cell lymphomas.
[Nature Medicine]

Abstract

Nanoparticle-Encapsulated
siRNAs for Gene Silencing in the Hematopoietic Stem-Cell Niche

Researchers report the design and in vivo performance of systemically injected lipid–polymer nanoparticles encapsulating small interfering RNA, for the silencing of genes in bone-marrow endothelial cells.
[Nature Biomedical Engineering]

Abstract

Itacitinib
(INCB039110), a JAK1 inhibitor, Reduces Cytokines Associated with Cytokine Release Syndrome Induced by CAR T-Cell Therapy

Investigators studied the effect of blocking JAK pathway signaling on CAR T-cell proliferation, anti-tumor activity and cytokine levels in in vitro and in vivo models. They report that itacitinib, a potent, selective JAK1 inhibitor, was able to significantly and dose-dependently reduce levels of multiple cytokines implicated in cytokine release syndrome in several in vitro and in vivo models.
[Clinical Cancer Research]

Full Article

Idelalisib
Treatment Prior to Allogeneic Stem Cell Transplantation for Patients with Chronic Lymphocytic Leukemia: A Report From the EBMT Chronic Malignancies Working Party

No studies have been reported so far on bridging treatment with idelalisib for patients with chronic lymphocytic leukemia prior to allogeneic hematopoietic cell transplantation. To study potential carry-over effects of idelalisib and to assess the impact of pathway-inhibitor failure researchers performed a retrospective EBMT registry-based study.
[Bone Marrow Transplantation]

Abstract

Impact
of Patient: Donor HLA Disparity on Reduced-Intensity-Conditioned Allogeneic Stem Cell Transplants from HLA Mismatched Unrelated Donors for AML: from the ALWP of the EBMT

Investigators studied 937 patients with acute myeloid leukemia in complete remission transplanted using a reduced intensity conditioning regimen from an unrelated donor mismatched at a single allele.
[Bone Marrow Transplantation]

Abstract

Novel 199 Base Pair NEFH
Promoter Drives Expression in Retinal Ganglion Cells

Researchers analysed EGFP expression driven by various sequences of the putative human NEFH promoter in order to define sequences required for preferential expression in retinal ganglion cells.
[Scientific Reports]

Full Article

Therapeutic
Angiogenesis Using Autologous Adipose-Derived Regenerative Cells in Patients with Critical Limb Ischaemia in Japan: A Clinical Pilot Study

Adipose tissue was obtained by ordinary liposuction method. Isolated adipose-derived regenerative cells (ADRCs) were injected into the ischaemic limb. Scientists performed therapeutic angiogenesis by cell transplantation-ADRC procedure in five patients with critical limb ischaemia.
[Scientific Reports]

Full Article

A
Defined N6-Methyladenosine (m⁶A) Profile Conferred by METTL3 Regulates Muscle Stem Cell/Myoblast State Transitions

RNAi studies showed that reducing levels of METTL3, the active m⁶A methyltransferase, reduced global m⁶A levels and forced C2C12 myoblasts to prematurely differentiate.
[Cell Death Discovery]

Full Article

Topologically
Selective Islet Vulnerability and Self-Sustained Downregulation of Markers for β-Cell Maturity in Streptozotocin-Induced Diabetes

Researchers showed that hyperglycemic Streptozotocin-treated mice still harbor a large pool of remaining β-cells but displayed pancreas-wide downregulation of glucose transporter type 2. Islet gene expression studies confirmed this downregulation and revealed impaired β-cell maturity.
[Communications Biology]

Full Article

Unexpected
Mutual Regulation Underlies Paralogue Functional Diversification and Promotes Epithelial Tissue Maturation in Tribolium

Scientists dissected the paralogues’ expression dynamics and transcriptional targets throughout embryogenesis. They identified an unexpected role of Tc-Zen2 in repression of Tc-zen1, generating a negative feedback loop that promoted developmental progression.
[Communications Biology]

Full Article

Resources for Your T Cell Therapy Research

REVIEWS

Modulating Gene Regulation to Treat Genetic Disorders

Scientists review emerging cis-regulation therapy (CRT) technologies and assess their therapeutic potential for treating a wide range of diseases caused by abnormal gene dosage. The challenges facing the translation of CRT into the clinic are discussed.
[Nature Reviews Drug Discovery]

Abstract

Clinical Development of CAR T Cell Therapy in China: 2020 Update

The authors systematically summarize the clinical practice of CAR T-cell therapy in China. They provide an informative reference for colleagues in the field, and a better understanding of the history and current situation will help researchers more reasonably conduct research and promote cooperation.
[Cellular & Molecular Immunology]

Full Article

INDUSTRY AND POLICY NEWS

bluebird
bio Announces European Medicines Agency’s Acceptance of Marketing Authorization Application (MAA) for elivaldogene autotemcel (eli-cel, Lenti-D™) Gene Therapy for Cerebral Adrenoleukodystrophy (CALD)

bluebird bio, Inc. announced that the European Medicines Agency accepted the company’s marketing authorization application for its investigational elivaldogene autotemcel gene therapy for the treatment of patients with cerebral adrenoleukodystrophy.
[bluebird bio, Inc.]

Press Release

Vor
Biopharma and Arbor Biotechnologies to Collaborate on Engineered Hematopoietic Stem-Cell Therapies

Vor Biopharma and Arbor Biotechnologies announced an agreement to use Arbor’s gene editing technologies to engineer hematopoietic stem cells, towards the goal of developing therapies for the treatment of blood cancers, such as acute myeloid leukemia.
[Vor Biopharma]

Press Release

Sarepta Therapeutics Investigational Gene Therapy SRP-9003 for the Treatment of Limb-Girdle Muscular Dystrophy Type 2E Shows Sustained Functional Improvements 18-months after Administration

Sarepta Therapeutics, Inc. announced positive results from the ongoing study of SRP-9003, the company’s investigational gene therapy for limb-girdle muscular dystrophy Type 2E.
[Sarepta Therapeutics, Inc. (GlobeNewswire, Inc.)]

Press Release

AVROBIO
Receives Orphan Drug Designation from the European Medicines Agency for AVR-RD-02, an Investigational Gene Therapy for Gaucher Disease

AVROBIO, Inc. announced that the European Commission has granted orphan drug designation for AVR-RD-02, the company’s investigational gene therapy for the treatment of Gaucher disease.
[AVROBIO, Inc.]

Press Release

FEATURED EVENT

Bispecific
Anti-CD20, Anti-CD19 CAR T Cells for Relapsed B Cell Malignancies: A Phase I Dose Escalation and Expansion Trial

Characteristics
of Anti-CD19 CAR T Cell Infusion Products Associated with Efficacy and Toxicity in Patients with Large B Cell Lymphomas

Nanoparticle-Encapsulated
siRNAs for Gene Silencing in the Hematopoietic Stem-Cell Niche

Itacitinib
(INCB039110), a JAK1 inhibitor, Reduces Cytokines Associated with Cytokine Release Syndrome Induced by CAR T-Cell Therapy

Idelalisib
Treatment Prior to Allogeneic Stem Cell Transplantation for Patients with Chronic Lymphocytic Leukemia: A Report From the EBMT Chronic Malignancies Working Party

Impact
of Patient: Donor HLA Disparity on Reduced-Intensity-Conditioned Allogeneic Stem Cell Transplants from HLA Mismatched Unrelated Donors for AML: from the ALWP of the EBMT

Novel 199 Base Pair NEFH
Promoter Drives Expression in Retinal Ganglion Cells

Therapeutic
Angiogenesis Using Autologous Adipose-Derived Regenerative Cells in Patients with Critical Limb Ischaemia in Japan: A Clinical Pilot Study

A
Defined N6-Methyladenosine (m⁶A) Profile Conferred by METTL3 Regulates Muscle Stem Cell/Myoblast State Transitions

Topologically
Selective Islet Vulnerability and Self-Sustained Downregulation of Markers for β-Cell Maturity in Streptozotocin-Induced Diabetes

Unexpected
Mutual Regulation Underlies Paralogue Functional Diversification and Promotes Epithelial Tissue Maturation in Tribolium

Modulating Gene Regulation to Treat Genetic Disorders

Clinical Development of CAR T Cell Therapy in China: 2020 Update

bluebird
bio Announces European Medicines Agency’s Acceptance of Marketing Authorization Application (MAA) for elivaldogene autotemcel (eli-cel, Lenti-D™) Gene Therapy for Cerebral Adrenoleukodystrophy (CALD)

Vor
Biopharma and Arbor Biotechnologies to Collaborate on Engineered Hematopoietic Stem-Cell Therapies

Sarepta Therapeutics Investigational Gene Therapy SRP-9003 for the Treatment of Limb-Girdle Muscular Dystrophy Type 2E Shows Sustained Functional Improvements 18-months after Administration

AVROBIO
Receives Orphan Drug Designation from the European Medicines Agency for AVR-RD-02, an Investigational Gene Therapy for Gaucher Disease

CAR-T Beyond Oncology Digital Event

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Research Fellow – Immunotherapy Development

Vice
President – Oncology and Cell Therapy

Postdoctoral Scholar – Biomedical Engineering in Regenerative Medicine

Research
Positions – Cancer and Immunotherapy

Research
Fellow Position – Neural Cell Therapy

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