Cell Therapy News Volume 20.30 | Sep 9 2019

TOP STORY

A Biodegradable Nanocapsule Delivers a Cas9 Ribonucleoprotein Complex for In Vivo Genome Editing

Researchers report the design of customizable synthetic nanoparticles for the delivery of Cas9 nuclease and a single-guide RNA that enabled the controlled stoichiometry of CRISPR components and limited the possible safety concerns in vivo. [Nat Nanotechnol] Abstract | Press Release

(Ranked by impact factor of the journal)

Enhanced CAR T Cell Expansion and Prolonged Persistence in Pediatric Patients with ALL Treated with a Low-Affinity CD19 CAR

In a clinical study, 12/14 patients with relapsed/refractory pediatric B cell acute lymphoblastic leukemia (ALL) treated with CD19 chimeric antigen receptor (CAR) T cells achieved molecular remission. [Nat Med] Abstract

Successful Engraftment of Gene-Corrected Hematopoietic Stem Cells in Non-Conditioned Patients with Fanconi Anemia

Researchers demonstrated that lentiviral-mediated hematopoietic gene therapy reproducibly conferred engraftment and proliferation advantages of gene-corrected hematopoietic stem cells in non-conditioned patients with Fanconi anemia subtype A. [Nat Med] Abstract

Development of Hematopoietic Stem Cell-Engineered Invariant Natural Killer T Cell Therapy for Cancer

Using a human hematopoietic stem cell (HSC) engrafted mouse model and a human invariant natural killer T (iNKT) TCR gene engineering approach, scientists demonstrated the efficient and long-term generation of HSC-iNKT cells in vivo. [Cell Stem Cell] Abstract | Graphical Abstract

Human Genome-Edited Hematopoietic Stem Cells Phenotypically Correct Mucopolysaccharidosis Type I

Investigators present an efficient ex vivo genome editing approach using CRISPR-Cas9 that targeted the lysosomal enzyme iduronidase to the CCR5 safe harbor locus in human CD34⁺ hematopoietic stem and progenitor cells. [Nat Commun] Full Article

Targeted Delivery of CRISPR Interference System against Fabp4 to White Adipocytes Ameliorates Obesity, Inflammation, Hepatic Steatosis, and Insulin Resistance

CRISPR interference mechanism based on catalytically dead Cas9 and single guide RNA was combined with a targeted nonviral gene delivery system to treat obesity and obesity-induced type 2 diabetes. [Genome Res] Abstract | Editorial

Therapeutic Genome Editing of Triple-Negative Breast Tumors Using a Noncationic and Deformable Nanolipogel

The loss of Lcn2 significantly inhibited the migration and the mesenchymal phenotype of human triple-negative breast cancer (TNBC) cells and subsequently attenuated TNBC aggressiveness. [Proc Natl Acad Sci USA] Abstract

Fetal Gene Therapy Using Single Injection of Recombinant AAV9 Rescued SMA Phenotype in Mouse

Mouse fetuses received a single intrauterine-intracerebroventricular injection of a single stranded or self-complementary (sc) adeno associated virus (AAV)9-survival motor neuron vector that led to 93 or 171 days lifespan of spinal muscular atrophy (SMA) mice. The muscle pathology and number of the motor neurons also improved in both study groups, with slightly better results coming from scAAV treatment. [Mol Ther] Abstract

Gene Correction of LGMD2A Patient-Specific iPSCs for the Development of Targeted Autologous Cell Therapy

Transplantation of gene-corrected limb girdle muscular dystrophy type 2A (LGMD2A) myogenic progenitors in a novel mouse model combining immunodeficiency and a lack of calpain 3 (CAPN3) resulted in muscle engraftment and rescue of the CAPN3 mRNA. [Mol Ther] Abstract

CRISPR-Cas9 Gene Editing in Lizards through Microinjection of Unfertilized Oocytes

Scientists demonstrated that the microinjection of immature oocytes within the ovaries of Anolis sagrei females enabled the production of CRISPR-Cas9-induced mutations. This method was capable of producing F0 embryos and hatchlings with monoallelic or biallelic mutations. [Cell Rep] Full Article | Graphical Abstract

Gene Therapy Correction of Aldehyde Dehydrogenase 2 Deficiency

Following acute ethanol ingestion, untreated ALDH2-deficient mice had elevated acetaldehyde levels and performed poorly in behavioral tests. In contrast, treated Aldh2^-/- and Aldh2^E487K+/+ mice had lower serum acetaldehyde levels and improved behavior. [Mol Ther Methods Clin Dev] Abstract | Full Article

AAV Gene Therapy Utilizing Glycosylation-Independent Lysosomal Targeting Tagged GAA in the Hypoglossal Motor System of Pompe Mice

Mice received an injection into the base of the tongue with adeno-associated virus serotype 9 (AAV9) encoding either the tagged or untagged enzyme; tissues were harvested four months later. Both AAV9 constructs effectively drove acid-alpha glucosidase (GAA) expression in lingual myofibers and hypoglossal motoneurons. [Mol Ther Methods Clin Dev] Abstract | Full Article

Direct Neuronal Reprogramming of Olfactory Ensheathing Cells for CNS Repair

Genome-wide RNA-sequencing analysis showed that the transcriptome profile of olfactory ensheathing cells (OECs) was effectively reprogrammed towards that of neuronal lineage. Importantly, these OEC-derived induced neurons survived and matured after transplantation into adult mouse spinal cords. [Cell Death Dis] Full Article

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Pluripotent Stem Cell-Based Therapeutics for Muscular Dystrophies

Researchers highlight the major research advances, challenges, and future prospects towards the development of pluripotent stem cell-based therapeutics for muscular dystrophy. [Trends Mol Med] Abstract

Transplantation of Human Embryonic Stem Cell-Derived Retinal Pigment Epithelial Cells (MA09-hRPE) in Macular Degeneration

Through in-depth, evidence-based analysis of the MA09-hRPE cell line, along with other human embryonic stem cell (hESC)-derived retinal pigment epithelium (RPE) cell lines, the author draws attention to the key technical challenges pertinent to the generation of a biologically competent hESC-RPE cell line and distill the four key prognostic factors residing in the host retina, which concurrently determine the outcomes of clinical efficacy and visual benefits. [npj Regen Med] Full Article

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The Wistar Institute and IMV Announce Research Collaboration to Develop New Targeted Immunotherapy against the Common BRAF Cancer Mutation

The Wistar Institute and IMV Inc. announced a collaboration to develop a targeted T-cell therapy against a common BRAF cancer mutation. [The Wistar Institute] Press Release

Celgene, Immatics Launch Up-to-$1.5B+ Cancer T Cell Collaboration

Celgene will partner with Immatics Biotechnologies to develop novel adoptive cell therapies against multiple cancers, through a collaboration that could generate more than $1.5 billion for the German T cell immunotherapy developer—and further expand Celgene’s oncology pipeline in advance of its planned $74 billion acquisition by Bristol-Myers Squibb. [Celgene (Genetic Engineering & Biotechnology News)] Press Release

Vertex to Acquire Semma Therapeutics with a Goal of Developing Curative Cell-Based Treatments for Type 1 Diabetes

Vertex Pharmaceuticals Incorporated announced that the company has entered into a definitive agreement under which Vertex will acquire Semma Therapeutics, a privately held biotechnology company pioneering the use of stem cell-derived human islets as a potentially curative treatment for type 1 diabetes. [Vertex Pharmaceuticals Incorporated] Press Release

Mustang Bio Announces License Agreement with CSL Behring for the Cytegrity^™ Stable Producer Cell Line for the Production of MB-107 Lentiviral Gene Therapy

Mustang Bio, Inc. announced that it has entered into a license agreement with CSL Behring for the Cytegrity^™ stable producer cell line developed and used by St. Jude Children’s Research Hospital. Cytegrity^™ stable producer cell line will be used to produce the viral vector for Mustang Bio’s MB-107 lentiviral gene therapy program for the treatment of X-linked severe combined immunodeficiency. [Mustang Bio, Inc.] Press Release

Adaptimmune and Noile-Immune Announce Agreement to Develop SPEAR T-Cell Products Expressing IL-7 and CCL19 as a Next-Generation Treatment for Cancer Patients

Adaptimmune Therapeutics plc and Noile-Immune Biotech, Inc. announced that they will co-develop next-generation SPEAR T-cell products, incorporating Noile-Immune’s PRIME technology, based upon co-expression of IL-7 and CCL19. [Adaptimmune Therapeutics plc] Press Release

Phio Pharmaceuticals Announces Research Collaboration with Carisma Therapeutics to Evaluate Its Self-Delivering RNAi Technology to Enhance Macrophage-Based Adoptive Cell Therapy

Phio Pharmaceuticals Corp. announced that it has entered into a research collaboration with Carisma Therapeutics to evaluate the potential of Phio’s self-delivering RNAi compounds to synergistically modify Carisma’s chimeric antigen receptor macrophages. [Phio Pharmaceuticals Corp.] Press Release

Generex Biotechnology Signs Letter of Intent to Acquire 51% in ALTuCELL, Inc. and Their Cellular Therapy Product Altsulin^® with Patented Microencapsulation Technology for the Treatment of Type I Diabetes Mellitis

Generex Biotechnology Corporation announced that the company has signed a binding letter of intent to partner with ALTuCELL. [Generex Biotechnology] Press Release

Woman Is First to Receive Cornea Made from ‘Reprogrammed’ Stem Cells

A Japanese woman in her forties has become the first person in the world to have her cornea repaired using reprogrammed stem cells. At a press conference, ophthalmologist Kohji Nishida from Osaka University, Japan, said the woman has a disease in which the stem cells that repair the cornea, a transparent layer that covers and protects the eye, are lost. [Nature News] Editorial

Krystal Biotech Announces Initiation of Phase I/II Study of KB105 in Transglutaminase-1 (TGM1) Deficient Autosomal Recessive Congenital Ichthyosis (ARCI)

Krystal Biotech Inc. announced the initiation of the Phase I/II study of KB105 in TGM1 deficient aARCI. The study, termed GEM-3 study, will examine KB105, an HSV-1 based gene therapy engineered to deliver a human TGM1 gene, in patients with ARCI. [Krystal Biotech Inc.] Press Release

uniQure Announces Achievement of Planned Enrollment in HOPE-B Pivotal Trial of AMT-061 (Etranacogene Dezaparvovec) in Patients with Hemophilia B

uniQure N.V. announced that the planned enrollment of 56 patients has been achieved in the HOPE-B pivotal trial of etranacogene dezaparvovec, an investigational AAV5-based gene therapy incorporating the patent-protected FIX-Padua variant for the treatment of patients with severe and moderately severe hemophilia B. [uniQure N.V.] Press Release

Sigilon Therapeutics Receives Orphan Drug Designation for SIG-001 for the Treatment of Hemophilia A

Sigilon Therapeutics announced it has received Orphan Drug Designation for SIG-001, an investigational therapy for hemophilia A that leverages Sigilon’s Shielded Living Therapeutics^™ platform to implant cells engineered to produce stable blood plasma levels of factor VIII, a crucial clotting protein. [Sigilon Therapeutics] Press Release

Fate Therapeutics Announces FDA Clearance of IND Application for FT596 Off-the-Shelf, iPSC-Derived CAR NK Cell Cancer Immunotherapy

Fate Therapeutics, Inc. announced that the FDA has cleared the company’s Investigational New Drug (IND) application for FT596, the company’s first off-the-shelf chimeric antigen receptor (CAR) natural killer (NK) cell cancer immunotherapy which targets multiple tumor-associated antigens. [Fate Therapeutics, Inc.] Press Release

Lineage Cell Therapeutics Announces Issuance of US Patent for Method of Treating Spinal Cord Injury with Stem Cell-Derived Oligodendrocyte Progenitor Cells

Lineage Cell Therapeutics, Inc. announced that the United States Patent and Trademark Office has issued U.S. Patent No. 10,286,009, entitled “Pluripotent stem cell-derived oligodendrocyte progenitor cells for the treatment of spinal cord injury” covering methods for utilizing pluripotent stem cell-derived oligodendrocyte progenitor cells for the treatment of spinal cord injury. [Lineage Cell Therapeutics, Inc.] Press Release

Alliance for Regenerative Medicine Issues Guiding Ethical Principles for Gene Editing

Ever since Chinese researcher He Jiankui announced he had used CRISPR-Cas9 gene editing to alter the DNA of embryos for seven couples, the international science community has been grappling with when it is and is not appropriate to use gene-editing techniques. [BioSpace] Editorial

Battleground Narrowed in CRISPR Patent Case between University of California and Broad

The contours of the latest battle over patents on the CRISPR genome editing technology are becoming a little clearer: In a decision released Monday evening, the US patent office told the Broad Institute, on one side, and the University of California and its allies, on the other, what they’ll be allowed to fight about. [STAT News] Editorial

NIH

Prospective Grant of an Exclusive Patent License: Genetically-Modified Lymphocytes for Cancer Therapy (FR Doc. No:2019-18648) Notice

Laboratory Animal Welfare: Report on Reducing Administrative Burden for Researchers: Animal Care and Use in Research (FR Doc. No:2019-18611) Notice

NEW SCSS – ISCT Joint Symposium 2019 “Frontiers in Cell Therapy”
November 13-15, 2019
Singapore, Singapore

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

NEW Postdoctoral Fellow – Cancer Research (Baylor College of Medicine)

NEW Postdoctoral Fellow – Molecular Genetic Basis of Behavioral Variation (University of California, San Francisco)

NEW Postdoctoral Fellow – Cancer Therapy (Purdue University)

NEW Postdoctoral Researcher – Cell Cycles in Stem Cells (University of California, Santa Cruz)

Research Technologist (STEMCELL Technologies Inc.)

Scientific Communications Coordinator (STEMCELL Technologies Inc.)

Project Leader – Gene Editing (San Raffaele Telethon Institute for Gene Therapy)

Postdoctoral Fellowship – Cancer Immunotherapy (McGill University)

Postdoctoral Position – Gene Therapy of Muscular Dystrophy (Ohio State University)

Postdoctoral Scholar – Gene and Cell Therapy (Stanford University)

Postdoctoral Scientist – HSV Vectors as a Therapy for Nervous System Tumors (Nationwide Children’s Hospital)

Research Scientist – Cellular Electrophysiology (Duke University)

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

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

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

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