Cell Therapy News Volume 20.22 | Jun 24 2019

TOP STORY

T Cell Receptor Gene Therapy Targeting WT1 Prevents Acute Myeloid Leukemia Relapse Post-Transplant

Scientists isolated a high-affinity Wilms’ Tumor Antigen 1-specific T cell expressing receptor (TCR_C4) from HLA-A2⁺ normal donor repertoires, inserted TCR_C4 into Epstein-Barr virus-specific donor CD8⁺ T cells to minimize graft-versus-host disease risk and enhance transferred T cell survival, and infused these cells prophylactically post-hematopoietic cell transplantation into 12 patients. [Nat Med] Abstract

(Ranked by impact factor of the journal)

Hematopoietic Stem Cell-Targeted Neonatal Gene Therapy with a Clinically Applicable Lentiviral Vector Corrects Osteopetrosis in oc/oc Mice

The authors showed that the osteopetrotic phenotype in oc/oc mice can be reversed by hematopoietic stem cell-targeted gene therapy with a clinically applicable lentiviral vector expressing a non-mutated form of human TCIRG1 under the mammalian promoter elongation factor 1α short. [Hum Gene Ther] Abstract

An Engineered Galactosylceramidase Construct Improves AAV Gene Therapy for Krabbe Disease in Twitcher Mice

To explore adeno-associated virus serotype 9 (AAV9) therapy for Krabbe disease, researchers engineered a codon-optimized AAV9 galactosylceramidase vector. They tested the optimized vector by a single systemic injection in the twitcher mouse, an authentic Krabbe disease model. [Hum Gene Ther] Abstract

The Impact of Re-Induction Prior to Salvage Autologous Stem Cell Transplantation in Multiple Myeloma

In multivariate models, re-induction did not affect time to next therapy (TNT)/overall survival (OS). In the subgroup of 188 patients who were re-induced, patients with relapsed/refractory disease at the time of autologous stem cell transplantation (ASCT) had significantly shorter TNT/OS compared to patients with deeper pre-ASCT responses. [Bone Marrow Transplant] Abstract

Traceless Aptamer-Mediated Isolation of CD8⁺ T Cells for Chimeric Antigen Receptor T-Cell Therapy

Investigators showed that chimeric antigen receptor T cells manufactured from these cells were comparable to antibody-isolated chimeric antigen receptor T cells in proliferation, phenotype, effector function and antitumor activity in a mouse model of B-cell lymphoma. [Nat Biomed Eng] Abstract

Transplantation of Human Embryonic Stem Cell Derived Retinal Tissue in the Subretinal Space of the Cat Eye

The authors successfully delivered human embryonic stem cell-derived retinal tissue into the subretinal space of the cat eye. They observed strong infiltration of immune cells in the graft and surrounding tissue in the cats treated with prednisolone. [Stem Cells Dev] Abstract

Successful Allogenic Stem Cell Transplantation in Patients with Inherited CARD9 Deficiency

Researchers report two unrelated patients from Brazil and Morocco with autosomal recessive caspase recruitment domain-containing protein 9 (CARD9) deficiency, both successfully treated with hematopoietic stem cell transplantation. [J Clin Immunol] Abstract

AAV-Mediated Neurotrophin Gene Therapy Promotes Improved Survival of Cochlear Spiral Ganglion Neurons in Neonatally Deafened Cats: Comparison of AAV2-hBDNF and AAV5-hGDNF

The authors explored the potential for two adeno-associated viral vectors (AAV) to elicit targeted neurotrophic factor expression in the cochlea and promote improved spiral ganglion and radial nerve fiber survival. [J Assoc Res Otolaryngol] Abstract

Personalized Pharmacokinetic Targeting with Busulfan in Allogeneic Hematopoietic Stem Cell Transplantation in Infants with Acute Lymphoblastic Leukemia

Investigators report busulfan pharmacokinetic data on hematopoietic stem cell transplantation conditioning for infants with KMT2A-gene-rearrangement-positive acute lymphoblastic leukemia. [Int J Hematol] Abstract

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Mesenchymal Stem Cells: Cell Therapy and Regeneration Potential

Researchers discuss and compare the characteristics of mesenchymal stromal/stem cells, and suggest that the variations in their distinctive features are dependent on the source and method of isolation as well as epigenetic changes during maintenance and growth. [J Tissue Eng Regen Med] Abstract

Transplantation of Stem Cells from Umbilical Cord Blood as Therapy for Type I Diabetes

Researchers compare the general characteristics of umbilical cord blood-derived multipotent stem cells and umbilical cord blood-derived mesenchymal stem cells and introduce their applications in type I diabetes mellitus. [Cell Tissue Res] Abstract

Translatable Gene Therapy for Lung Cancer Using Crispr CAS9—An Exploratory Review

The authors highlight important studies and findings in the perspective of lung cancer with an in-depth analysis of various clinical trials associated with the use of CRISPR Cas9 technology in the field of cancer research. [Cancer Gene Ther] Abstract

Visit our reviews page to see a complete list of reviews in the cell therapy research field.

Axovant and Yposkesi Sign Strategic Gene Therapy Development and Manufacturing Partnership

Axovant Gene Therapies Ltd. announced it has signed a strategic partnership with Yposkesi for preferred access and reserved capacity for cGMP grade viral vector production. [Axovant Gene Therapies Ltd.] Press Release

Gamida Cell and Lonza Establish Commercial Manufacturing Agreement for Omidubicel

Gamida Cell Ltd. and Lonza announced that the companies have entered into a strategic manufacturing agreement. [Gamida Cell Ltd.] Press Release

Krystal Biotech Announces Positive Results from Phase II Clinical Trial of KB103 and Receives Regenerative Medicine Advanced Therapy Designation from FDA for KB103

Krystal Biotech, Inc. announced positive results from its Phase II placebo-controlled clinical trial of KB103 study and an update on results from Phase I study. [Krystal Biotech, Inc.] Press Release

Arbutus Receives Regulatory Clearance to Initiate Phase Ia/Ib Clinical Trial of AB-729

Arbutus Biopharma Corporation announced it has received regulatory clearance to initiate a Phase Ia/Ib clinical trial of AB-729, Arbutus’ subcutaneously-administered RNA interference agent. [Arbutus Biopharma Corporation] Press Release

NantKwest Launches First-in-Class, First-in-Human Phase I Clinical Trial with a Targeted PD-L1 t-haNK Cell Therapy in Patients with Solid Tumors

NantKwest Inc. announced that the company’s PD-L1 t-haNK investigational new drug application has cleared FDA review and the program has now transitioned to a first-in-human clinical trial in patients with locally advanced or metastatic solid cancers. [NantKwest Inc.] Press Release

Rocket Pharmaceuticals Announces Patient Dosing Has Commenced for Phase I Clinical Trial of RP-A501, the First Gene Therapy to Treat a Monogenic Heart Failure Syndrome

Rocket Pharmaceuticals, Inc. announced that patient dosing has commenced in the open-label, Phase I clinical trial of RP-A501, the company’s adeno-associated viral vector-based gene therapy for the treatment of Danon disease. [Rocket Pharmaceuticals, Inc.] Press Release

Henry Ford Cancer Institute Treats Its First Patient with Innovative ‘Living Drug’ Therapy

Henry Ford Cancer Institute has treated its first patient with CAR T-cell therapy, an approach that uses engineered cells from a patient’s immune system to destroy cancer. [Henry Ford Cancer Institute] Press Release

Mesoblast Heart Failure Cell Therapy Receives Orphan Drug Designation from FDA for Prevention of Gastrointestinal Bleeding in Patients with Left Ventricular Assist Devices

Mesoblast Limited announced that the FDA has granted the company’s product candidate, rexlemestrocel-L, Orphan Drug Designation for prevention of post-implantation mucosal bleeding in end-stage chronic heart failure patients who require a left ventricular assist device. [Mesoblast Limited] Press Release

Incysus Therapeutics Announces Publication of US Patent for Drug Resistant Immunotherapy (DRI) Technology

Incysus Therapeutics, Inc. announced that the United States Patent and Trademark Office has issued US Patent No. 10,322,145 titled “Drug Resistant Immunotherapy for Treatment of a Cancer.” [Incysus Therapeutics, Inc. (GlobeNewswire, Inc.)] Press Release

CARsgen Therapeutics Receives IND Clearance for BCMA-CAR-T Cells from the US FDA

CARsgen Therapeutics announced that one of its leading drug candidates, CT053 fully human BCMA (B-Cell Maturation Antigen)-CAR-T cell for the treatment of patients suffering from relapsed/refractory multiple myeloma, has received Investigational New Drug (IND) clearance from the FDA. [CARsgen Therapeutics] Press Release

FDA Grants Fast Track Designation for Torque’s First Deep-Primed T Cell Cancer Immunotherapy Program, TRQ-1501

Torque announced that the FDA granted Fast Track designation for Torque’s first Deep-Primed™ T cell immunotherapy program, TRQ-1501. [Torque Therapeutics] Press Release

Triumvira Immunologics Announces Clearance of IND and CTA by US FDA and Health Canada for Its First TAC T-Cell Therapeutic Product Candidate, TAC01-CD19

Triumvira Immunologics, Inc. announced that both the FDA and Health Canada have cleared Triumvira’s Investigational New Drug (IND) and Clinical Trial Applications (CTA) for its novel T cell therapy product TAC01-CD19 in patients with CD19-postive B-cell malignancies. [Triumvira Immunologics, Inc.] Press Release

Abeona Therapeutics Receives FDA Fast Track Designation for ABO-202 AAV9 Gene Therapy in CLN1 Disease

Abeona Therapeutics Inc. announced that the FDA has granted Fast Track designation to its ABO-202 program. [Abeona Therapeutics Inc.] Press Release

Alarmed by New ‘CRISPR Babies’ Plan, Top Science Figures Say They’re Powerless to Stop It

Two influential leaders in science for the first time publicly condemned a Russian biologist who said he plans to produce gene-edited babies but conceded that it was beyond their organizations’ authority to halt him from doing so. [STAT News] Editorial

Iranian Biologists Face US Trial for Trying to Take Proteins Out of the Country

Three Iranian biologists are facing trial in a US court over charges that they violated trade sanctions by attempting to export chemicals known as growth factors, which are commonly used in medical research. The scientists have asked a federal district court judge in Atlanta, Georgia, to dismiss the criminal charges against them. [Nature News] Editorial

NEW Cell Symposia: ‘Gene and Cell Based Therapies: CRISPR, Stem Cells & Beyond’
March 2-4, 2020
San Francisco, California, United States

Visit our events page to see a complete list of events in the community.

NEW Senior Scientist – Immuno-Oncology of Hematologic Malignancies (University of Kansas School of Medicine)

NEW Research Scientist – Cellular Electrophysiology (Duke University)

Stem Cell Podcast Co-Host (Stem Cell Podcast)

Postdoctoral Scientist – Computational Biologist (Cedars-Sinai Medical Center)

Senior Scientist – Antigen Presentation (Astellas Pharma)

Postdoctoral Research Fellowships – Kidney Disease (Vanderbilt University Medical Center)

Postdoctoral Scholarship – Gene Therapy for Osteopetrosis (Lund University)

Postdoctoral Fellow – Hematopoietic Stem Cells and Gene Therapy (UC San Diego)

Postdoctoral Position – Biomedical Engineering (US Food and Drug Administration)

Postdoctoral Research Fellow – Translational Gene Therapy (Fred Hutchinson Cancer Research Center)

Director of Research – Division of Translational Medicine & Human Genetics (University of Pennsylvania)

Cell Therapy Associate III – Cellular Therapeutics (KBI Biopharma)

Postdoctoral Fellow – Single Cell Genomics (BC Cancer Research Center)

Tenure Track Faculty Position – Cancer Biology & Therapeutics (Geisel School of Medicine at Dartmouth)

Postdoctoral Fellowship – Natural Killer Cells, Cancer and Immunotherapy (Dalhousie University)

Assistant/Associate or Professor – Cancer Immunotherapy/Tumor Immunology (University of California, Irvine)

Faculty Positions – Hematology and Oncology (University of California, Davis)

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