Cell Therapy News Volume 19.23 | Jul 9 2018

TOP STORY

Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis

In a Phase III trial, the authors randomly assigned patients with hereditary transthyretin amyloidosis with polyneuropathy, in a 2:1 ratio, to receive intravenous patisiran or placebo once every three weeks. Patisiran improved multiple clinical manifestations of hereditary transthyretin amyloidosis. [N Engl J Med] Abstract

(Ranked by impact factor of the journal)

Human Embryonic Stem Cell-Derived Cardiomyocytes Restore Function in Infarcted Hearts of Non-Human Primates

Scientists showed that transplantation of ~750 million cryopreserved human embryonic stem cell-derived cardiomyocytes enhanced cardiac function in macaque monkeys with large myocardial infarctions. [Nat Biotechnol] Abstract | Press Release

Efficient Gene Transfer to Kidney Mesenchymal Cells Using a Synthetic Adeno-Associated Viral Vector

Investigators evaluated the transduction profiles of various pseudotyped adeno-associated virus vectors expressing either GFP or Cre recombinase reporters in mouse kidney and human kidney organoids. [J Am Soc Nephrol] Abstract | Press Release | Graphical Abstract

Novel Lineage Depletion Preserves Autologous Blood Stem Cells for Gene Therapy of Fanconi Anemia Complementation Group A

Transduced lineage- cell products engrafted equivalently to that of purified CD34⁺ cells from the same donor when xenotransplanted at matched CD34⁺ cell doses. [Haematologica] Abstract | Full Article

In Vivo Tracking and 1H/19F MRI of Biodegradable Polyhydroxyalkanoate/Polycaprolactone Blend Scaffolds Seeded with Labeled Cardiac Stem Cells

Seeded scaffolds were implanted and studied in the post-mortem murine heart in situ, and in two additional C57BL/6 mice in vivo and imaged immediately after and at seven days post-implantation. [ACS Appl Mater Interfaces] Abstract

An Immune System-Modified Rat Model for Human Stem Cell Transplantation Research

The authors generated a human SIRPα ⁺Prdkc^−/−Il2rγ^−/− rat model, denoted NODSCIDIl2rγ^−/−-like rat, which expresses human SIRPα and is abolished in the development of B, T, and natural killer cells. [Stem Cell Reports] Full Article

AAV5-miHTT Gene Therapy Demonstrates Broad Distribution and Strong Human Mutant Huntingtin Lowering in a Huntington Disease Minipig Model

Scientists investigated the feasibility, efficacy, and tolerability of one-time intracranial administration of microRNA targeting human huntingtin, delivered via adeno-associated viral vector serotype 5 (AAV5-miHTT) in the transgenic Huntington disease minipig model. [Mol Ther] Abstract | Full Article

Retinal AAV8-RS1 Gene Therapy for X-Linked Retinoschisis: Initial Findings from a Phase I/IIa Trial by Intravitreal Delivery

Researchers evaluated the safety and tolerability of ocular RS1 adeno-associated virus (AAV8-RS1) gene augmentation therapy to the retina of participants with X-linked retinoschisis. [Mol Ther] Abstract

Hypoxic Preconditioning of Mesenchymal Stem Cells with Subsequent Spheroid Formation Accelerates Repair of Segmental Bone Defects

PC3 spheroids were transplanted into rat critical-sized femoral segmental defects to evaluate their potential for bone healing. Spheroid-containing gels induced significantly more bone healing compared with gels containing preconditioned individual mesenchymal stem cells or acellular gels. [Stem Cells] Full Article

Stem Cell Induced Pulp Regeneration Can Be Enhanced by Administration of CCL11 Neutralizing Antibody in the Ectopic Tooth Transplantation Model in the Aged Mice

MDPSC transplants were determined in an ectopic tooth root transplantation model in both the aged and young mice. The amount of regenerated pulp tissue was analyzed in the transplants with continuous administration of CCL11 antibody compared with those without the antibody administration. [Rejuvenation Res] Abstract

A 12-Month Follow-Up after a Single Intracavernous Injection of Autologous Adipose-Derived Regenerative Cells in Patients with Erectile Dysfunction following Radical Prostatectomy: An Open-Label Phase I Clinical Trial

Twenty-one patients with ED after radical prostatectomy, with no signs of recovery using conventional therapy, received a single intracavernous injection of autologous adipose-derived regenerative cells and were followed for one year. [Urology] Abstract | Press Release

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Chimeric Antigen Receptor Therapy

Investigators address T-cell engineering and synthetic immunity, with a focus on producing durable remissions in patients with treatment-refractory tumors. [N Engl J Med] Abstract

Gene Therapy Strategies in the Treatment of Hypertrophic Cardiomyopathy

The authors summarize recent technological advances and their implications as gene therapy options in hypertrophic cardiomyopathy with a special focus on treating MYBPC3 mutations and its potential for being a successful bench to bedside example. [Pflugers Arch] Abstract

Targeting Corneal Inflammation by Gene Therapy: Emerging Strategies for Keratitis

Researchers focus on the progress and challenges of gene therapy to treat corneal inflammation. After introducing the inflammation process, they present the main nucleic acid delivery systems, including viral and non-viral vectors, and the most studied strategies to address the therapy: control of neovascularization and regulation of pro- and anti-inflammatory cytokines. [Exp Eye Res] Abstract | Graphical Abstract

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

Immunotherapy Pioneer and Serial Biotech Entrepreneur Patrick Baeuerle to Present Compelling Data from Harpoon Therapeutics’ Biotherapeutics

Patrick Baeuerle, Ph.D. will give a keynote presentation about next-generation immuno-oncology therapies from Harpoon Therapeutics, Inc. [Press release from Harpoon Therapeutics (BusinessWire, Inc.) discussing research to be presented at the 2018 European Congress on Biotechnology (ECB), Geneva] Press Release

Cellect Signs Collaboration Agreement with Dresden University denovoMATRIX Team

Cellect Biotechnology Ltd. announced that it has signed a collaboration and material transfer agreement with the denovoMATRIX group of the Technische Universität Dresden. [Cellect Biotechnology Ltd.] Press Release

TxCell Exercises In-License Option to the University of British Columbia’s Intellectual Property Rights of the HLA-A2 CAR-Treg Program

TxCell SA announced it has exercised its exclusive option to in-license the intellectual property rights of one of its academic partners, the University of British Columbia, onto the HLA-A2 CAR-regulatory T cell (Treg) program. [TxCell SA] Press Release

uniQure Enrolls First Patient in Phase III HOPE-B Pivotal Study of AMT-061 in Patients with Hemophilia B

uniQure N.V. announced that it has enrolled its first patient in the Phase III HOPE-B pivotal study of AMT-061, an investigational AAV5-based gene therapy incorporating the FIX-Padua variant for the treatment of patients with severe and moderately severe hemophilia B. [uniQure N.V.] Press Release

Ziopharm Oncology Enrolls First Patient in Phase I Trial Evaluating Combination Therapy of Controlled IL-12 and OPDIVO^®

Ziopharm Oncology, Inc. announced that it has enrolled the first patient in its Phase I clinical trial to evaluate Ad-RTS-hIL-12 plus veledimex in combination with OPDIVO^®, an immune checkpoint, or PD-1, inhibitor, in adult patients with recurrent glioblastoma. [Ziopharm Oncology, Inc.] Press Release

Cellerant Therapeutics, Inc. Announces FDA Grants Regenerative Medicine Advanced Therapy (RMAT) Designation for Romyelocel-L to Prevent Infections during Neutropenia

Cellerant Therapeutics, Inc. announced that the FDA has granted RMAT designation for romyelocel-L for the prevention of serious bacterial and fungal infections in patients with de novo acute myeloid leukemia undergoing induction chemotherapy. [Cellerant Therapeutics, Inc.] Press Release

BioTime Awarded Grant from the NIH

BioTime, Inc. announced that it has been awarded a grant of $743,345 from the Small Business Innovation Research program of the National Institutes of Health (NIH). [BioTime, Inc.] Press Release

Kolon TissueGene to Start US Phase III Clinical Trial for Invossa™

Kolon TissueGene, Inc. announced that Clinical Hold issued by the FDA has been lifted effective July 5, 2018 and that the company now has FDA Approval to move forward with its clinical trials involving patients diagnosed with knee osteoarthritis. [Kolon TissueGene, Inc.] Press Release

BrainStorm Granted Japanese Patent for NurOwn^®

BrainStorm Cell Therapeutics Inc. announced that it has received a Decision to Grant notice from the Japanese Patent Office to issue a patent entitled: “Methods of generating mesenchymal stem cells which secrete neurotrophic factors.” [BrainStorm Cell Therapeutics Inc.] Press Release

Controversial CRISPR ‘Gene Drives’ Tested in Mammals for the First Time

A controversial technology capable of altering the genomes of entire species has been applied to mammals for the first time. In a preprint posted to bioRxiv on 4 July, researchers describe developing ‘gene drives’ — which could be used to eradicate problematic animal populations — in lab mice using the CRISPR gene-editing technique. [Nature News] Editorial

Germany’s Prestigious Max Planck Society Investigates New Allegations of Abuse

The Max Planck Society, one of Germany’s wealthiest and most prestigious research organizations, is investigating fresh allegations of bullying and sexual harassment following an anonymous survey at one of its institutes. [Nature News] Editorial

FDA

Revised Recommendations for Reducing the Risk of Zika Virus Transmission by Blood and Blood Components; Guidance for Industry; Availability (FR Doc. No:2018-14537) Notice

NIH

Prospective Grant of an Exclusive Patent License: Development of an Anti-Mesothelin Chimeric Antigen Receptor (CAR) for the Treatment of Human Cancer (FR Doc. No:2018-13893) Notice

NEW ComBio 2018
September 23-26, 2018
Sydney, Australia

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

NEW Scientia PhD – Gene Therapy (University of New South Wales)

NEW Postdoctoral Fellow – DNA Damage Repair and Pediatric Sarcoma Therapy (University of Texas Health Science Center at San Antonio)

Project Leader – Gene and Cellular Therapy (CELLECTIS Inc.)

Postdoctoral Position – Corneal Diseases (University of California, Los Angeles)

PhD Student – Cellular Immunotherapy (Glycostem Therapeutics)

Research Associate/PhD Position – iPS Cell-Derived Neurons to Model Parkinson’s Disease (TU Dresden)

Postdoctoral Fellow – Cell and Gene Therapies for Cardiac Arrhythmias and Heart Failure (Duke University)

Postdoctoral Position – Human Pluripotent Stem Cell-Based Cell Therapy in Type 1 Diabetes (University of Copenhagen)

Scientist/Senior Scientist – Lentivirus Production (Obsidian Therapeutics)

Scientist/Senior Scientist – Bioinformatics and Data Sciences (Obsidian Therapeutics)

Postdoctoral Fellow – Immunology, Immunotherapy, Gene Therapy (City of Hope)

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