Cell Therapy News Volume 20.29 | Aug 26 2019

TOP STORY

Adeno-Associated Viral Vector Serotype 9-Based Gene Therapy for Niemann-Pick Disease Type A

Researchers evaluated the safety of cerebellomedullary (CM) cistern injection of adeno-associated viral vector serotype 9 encoding human sphingomyelinase in nonhuman primates (NHP). They found that CM injection in NHP resulted in widespread transgene expression within brain and spinal cord cells without signs of toxicity. [Sci Transl Med] Abstract

(Ranked by impact factor of the journal)

Precise In Vivo Genome Editing via Single Homology ARM Donor Mediated Intron-Targeting Gene Integration for Genetic Disease Correction

The authors describe a versatile in vivo gene knock-in methodology that enabled the targeting of a broad range of mutations and cell types through the insertion of a minigene at an intron of the target gene locus using an intracellularly linearized single homology arm donor. [Cell Res] Abstract

Self-Organized Formation of Developing Appendages from Murine Pluripotent Stem Cells

Scientists report means for induction of an limb bud-like mesenchymal/epithelial complex tissues from murine pluripotent stem cells in vitro. They demonstrated that the induced tissues contributed to endogenous digit tissue after transplantation. [Nat Commun] Full Article

Human Skin-Derived ABCB5⁺ Stem Cell Injection Improves Liver Disease Parameters in Mdr2KO Mice

Sixteen weeks old Mdr2KO mice were i.v. injected with 500,000 ABCB5⁺ cells using different experimental setups. The effects of cellular therapy on inflammation, fibrosis, apoptosis, and proliferation were analyzed in the collected liver tissues. [Arch Toxicol] Abstract

Intercellular Adhesion Molecule-1 Enhances the Therapeutic Effects of MSCs in a Dextran Sulfate Sodium-Induced Colitis Models by Promoting MSCs Homing to Murine Colons and Spleens

A single injection of mesenchymal stem cells (MSCs) promoted general recovery and reduced pathological damage in inflammatory bowel disease (IBD) mice. The in vivo distribution analysis results indicated that a higher number of intercellular adhesion molecule-1-overexpressing MSCs homed to the colon and spleen of IBD mice. [Stem Cell Res Ther] Full Article

Bicistronic Transfer of CDKN2A and p53 Culminates in Collaborative Killing of Human Lung Cancer Cells In Vitro and In Vivo

In a mouse model of in situ gene therapy, a single intratumoral treatment with the bicistronic vector conferred markedly inhibited tumor progression while the treatment with either CDKN2A or p53 alone only partially controlled tumor growth. [Gene Ther] Abstract

In Vitro and In Vivo Evaluation of Discogenic Cells, An Investigational Cell Therapy for Disc Degeneration

After subcutaneous implantation in a nude mouse model, human discogenic cells were found to have generated regions rich with extracellular matrix over the course of four months, with no signs of tumorigenicity. Intradiscal injection of human discogenic cells in a rabbit model of disc degeneration caused an increase in disc height and improvement of tissue architecture relative to control discs or injection of vehicle alone with no signs of toxicity. [Spine J] Abstract

Targeted Exon Skipping with AAV-Mediated Split Adenine Base Editors

Scientists demonstrated the application of CRISPR-Cas9 adenine deaminase base editors to disrupt the conserved adenine within splice acceptor sites for programmable exon skipping. [Cell Discov] Full Article

Mesenchyme-Free Expansion and Transplantation of Adult Alveolar Progenitor Cells: Steps toward Cell-Based Regenerative Therapies

Researchers developed several mesenchyme-free culture conditions that promoted growth of murine alveolar type-2 (AT2) organoids. Transplanting dissociated AT2 organoids into influenza-infected mice demonstrated that organoids engrafted and either proliferated as AT2 cells or unexpectedly adopted a basal cell-like fate associated with maladaptive regeneration. [NPJ Regen Med] Full Article | Press Release

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Adoptive Cell Therapy Using Engineered Natural Killer Cells

Investigators discuss advantages and potential drawbacks of using natural killer cells as a novel cellular therapy against hematologic malignancies, as well as strategies to further enhance their effector function. [Bone Marrow Transplant] Abstract

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NGen Funding to Transform Manufacturing of Critical Materials in Gene Therapies

NGen has approved Supercluster funding for a transformative $4.2 million project led by iVexSol Canada to develop an advanced manufacturing process for lentiviral vectors. These gene-editing reagents are critical components in the manufacturing processes of cell and gene therapies (CGTs), which have been successfully employed to fight cancer and repair a variety of rare and inherited genetic disorders in both children and adults. [NGen (STEMCELL Technologies Inc.)] Press Release

AskBio Acquires Synpromics Ltd. and Expands Its Gene Therapy Technology Portfolio

AskBio has acquired Synpromics, the leader in gene control synthetic promoter technology, bioinformatics and intelligent data-driven design that enables more precise cell targeting and gene expression. [AskBio (PR Newswire Association LLC.)] Press Release

Pfizer Invests Half Billion Dollars to Advance State-of-the-Art Gene Therapy Facility in Sanford, North Carolina

Pfizer announced an additional half billion dollar investment for the construction of its state-of-the-art gene therapy manufacturing facility in Sanford, North Carolina. [Pfizer] Press Release

Rocket Pharmaceuticals Announces Clearance from the Spanish Agency for Medicines and Health Products for the Phase II Registration-Enabling FANCOLEN-II Study of RP-L102 for Fanconi Anemia

Rocket Pharmaceuticals, Inc. announces clearance from the Spanish Agency for Medicines and Health Products to commence enrollment in the FANCOLEN-II Phase II registration-enabling study of RP-L102 for Fanconi Anemia in Spain. [Rocket Pharmaceuticals, Inc.] Press Release

Mustang Bio and ST. Jude Children’s Research Hospital Announce MB-107 Lentiviral Gene Therapy for X-Linked Severe Combined Immunodeficiency (XSCID) Granted Regenerative Medicine Advanced Therapy (RMAT) Designation from FDA

Mustang Bio, Inc. and St. Jude Children’s Research Hospital announced that MB-107, a lentiviral gene therapy for the treatment of XSCID, also known as bubble boy disease, has been granted the RMAT designation by the FDA. [Mustang Bio, Inc.] Press Release

FDA Accepts IND Application for Novel Immunotherapeutic for Prostate Cancer in Development by Calibr at Scripps Research

Calibr announced that the FDA has cleared its investigational new drug application (IND) for CCW702, a novel immunotherapeutic for the treatment of patients with metastatic castration-resistant prostate cancer. [Calibr] Press Release

DiscGenics Receives FDA Fast Track Designation for Cell Therapy for Disc Degeneration

DiscGenics, Inc. announced that the FDA has granted Fast Track designation for its investigational cell therapy, IDCT, currently being evaluated in regulator-allowed clinical trials in the US and Japan for the reduction in pain and disability associated with degenerative disc disease. [DiscGenics, Inc.] Press Release

Lineage Cell Therapeutics Announces Issuance of US Patent for System for Generating Immunogenic Dendritic Cells

Lineage Cell Therapeutics, Inc. announced that the United States Patent and Trademark Office has issued US Patent No. 10,344,262, for a system for production of antigen presenting dendritic cells from human embryonic stem cells. [Lineage Cell Therapeutics, Inc.] Press Release

UC Receives Its 11^th U.S. Patent for CRISPR-Cas9

The University of California (UC), the University of Vienna and Emmanuelle Charpentier received an 11^th US patent involving CRISPR-Cas9, further expanding the reach of UC’s patented technology relating to this revolutionary gene-editing tool. [The University of California] Press Release

As Brexit Nears, Britain’s Drugs, Devices and Pricing Regulators Seek the Exit

Firm details on exactly how the UK will regulate new medicines is still to be decided after it leaves the EU later this year (caveats on timing abound), but we now know that the regulator won’t be run by Dr. Ian Hudson, who is stepping down after six years in charge. [FierceBiotech] Editorial

Huge US Government Study to Offer Genetic Counseling

A US government study that aims to sequence the genomes of one million volunteers will partner with a genetic-counselling company to help participants understand their results. It will be the largest US government study to provide such a service. [Nature News] Editorial

NIH

Prospective Grant of an Exclusive Patent License: Development and Commercialization of CD19/CD22 Chimeric Antigen Receptor (CAR) Therapies for the Treatment of B-Cell Malignancies (FR Doc. No:2019-17866) Notice

NEW Tumor Heterogeneity, Plasticity, and Therapy
May 14-15, 2020
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NEW Project Leader – Gene Editing (San Raffaele Telethon Institute for Gene Therapy)

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Postdoctoral Fellowship – Cancer Immunotherapy (McGill University)

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

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

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

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