Cell Therapy News Volume 18.38 | Nov 20 2017

TOP STORY

Boy Is Given New Skin Thanks to Gene Therapy

Scientists successfully treated a child suffering from extensive skin damage using transplants derived from genetically modified stem cells. The boy was a so-called butterfly child: he suffered from epidermolysis bullosa, a genetic skin disease that had destroyed approximately 80 percent of his epidermis. [Press release from Ruhr-Universität Bochum discussing online prepublication in Nature] Press Release | Abstract

(Ranked by impact factor of the journal)

CD22-Targeted CAR T Cells Induce Remission in B-ALL that Is Naive or Resistant to CD19-Targeted CAR Immunotherapy

Investigators report results from a Phase I trial testing a new CD22-targeted CAR in 21 children and adults, including 17 who were previously treated with CD19-directed immunotherapy. [Nat Med] Abstract | Press Release | Editorial

PD-L1 Genetic Overexpression or Pharmacological Restoration in Hematopoietic Stem and Progenitor Cells Reverses Autoimmune Diabetes

Genetically engineered or pharmacologically modulated hematopoietic stem and progenitor cells overexpressing PD-L1 inhibited the autoimmune response in vitro, reverted diabetes in newly hyperglycemic nonobese diabetic (NOD) mice in vivo, and homed to the pancreas of hyperglycemic NOD mice. [Sci Transl Med] Abstract | Press Release

Cardiac Progenitor Cells Activated by Mitochondrial Delivery of Resveratrol Enhance the Survival of a Doxorubicin-Induced Cardiomyopathy Mouse Model via the Mitochondrial Activation of a Damaged Myocardium

Scientists evaluated the survival rate of doxorubicin treated mice, with or without the injection of mitochondrial drug delivery system (MITO) cells into the myocardium. The MITO cell transplanted mice lived significantly longer than the conventional cardiac progenitor cell transplanted ones. [J Control Release] Abstract | Graphical Abstract

DNA-Mediated Gene Therapy in a Mouse Model of Limb Girdle Muscular Dystrophy 2B

To achieve gene delivery of plasmids encoding dysferlin to hind limb muscles of dysferlin knockout mice, the authors used a vascular injection method that perfused naked plasmid DNA into all major muscle groups of the hind limb. [Mol Ther Methods Clin Dev] Full Article

Human PBMC-Transferred Murine MHC Class I/II-Deficient NOG Mice Enable Long-Term Evaluation of Human Immune Responses

Administration of human peripheral blood mononuclear cells (PBMCs) into newly developed murine MHC class I- and class II-deficient NOG mice showed sufficient engraftment of human immune cells with little sign of graft versus host disease. [Cell Mol Immunol] Abstract

AAV-Mediated Transcription Factor EB (TFEB) Gene Delivery Ameliorates Muscle Pathology and Function in the Murine Model of Pompe Disease

Researchers evaluated the effects of TFEB systemic delivery on muscle pathology and on functional performance, a primary measure of efficacy in a disorder like Pompe disease (PD). They treated one-month-old PD mice with an AAV2.9-MCK-TFEB vector. [Sci Rep] Full Article

Autologous Adipose-Derived Regenerative Cell Therapy Modulates Development of Hypertrophic Scarring in a Red Duroc Porcine Model

The authors assessed the influence of local delivery of adipose-derived regenerative cells immediately following deep-partial thickness cutaneous injury on hypertrophic scar development in the red Duroc porcine model. [Stem Cell Res Ther] Full Article | Press Release

sRAGE Prolonged Stem Cell Survival and Suppressed RAGE-Related Inflammatory Cell and T Lymphocyte Accumulations in an Alzheimer’s Disease Model

Investigators generated soluble RAGE (sRAGE) secreting mesenchymal stem cells (MSCs) and then injected either these MSCs or control MSCs with amyloid beta 1–42 into the entorhinal cortices of male Sprague Dawley rats. [Biochem Biophys Res Commun] Abstract

Cell Therapy in Stroke—Cautious Steps towards a Clinical Treatment

In the future, stroke patients may receive stem cell therapy as this has the potential to restore lost functions. However, the development of clinically deliverable therapy has been slower and more challenging than expected. Despite recommendations by STAIR and STEPS consortiums, there remain flaws in experimental studies such as lack of animals with comorbidities, inconsistent approaches to experimental design, and concurrent rehabilitation that might lead to a bias towards positive results. [Transl Stroke Res] Abstract

Cell Therapy for Diverse Central Nervous System Disorders: Inherited Metabolic Diseases and Autism

In the past few decades, hematopoietic stem cell transplantation has expanded to additional sources of donor cells, a wider range of indications, and the development of novel cell products. This trajectory has sparked a rapidly growing interest in the pursuit of innovative cell therapies to treat presently incurable diseases, including neurological conditions. [Pediatr Res] Abstract

3-D Bioprinting of Neural Tissue for Applications in Cell Therapy and Drug Screening

Current treatment options for degenerative diseases focus on physical rehabilitation or drug therapies, which temporarily mask the effects of cell damage, but quickly lose their efficacy. Cell therapies for the central nervous system remain an untapped market due to the complexity involved in growing neural tissues, controlling their differentiation, and protecting them from the hostile environment they meet upon implantation. [Front Bioeng Biotechnol] Full Article

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Significant Improvements Reported in Duchenne Muscular Dystrophy Patients Treated with Capricor’s Investigational Cell Therapy

Boys and young men in advanced stages of Duchenne muscular dystrophy experienced significant and sustained improvements in cardiac structure and function, as well as skeletal muscle function, following treatment with CAP-1002, the lead investigational therapy under development. [Press release from Capricor Therapeutics, Inc. discussing research presented at the American Heart Association Scientific Sessions 2017, Anaheim] Press Release

ITUS Enters CAR-T Research Alliance with Moffitt Cancer Center

ITUS Corporation announced that it has entered into a Cooperative Research and Development Agreement with Moffitt Cancer Center to advance toward human clinical testing a CAR-T technology aimed initially at ovarian cancer, and eventually additional solid tumors. [ITUS Corporation] Press Release

Three-Year Follow-Up Phase III Data Provide Additional Information on Efficacy, Durability and Safety of Investigational LUXTURNA™ (Voretigene Neparvovec) in Patients with Biallelic RPE65-Mediated Inherited Retinal Disease

Spark Therapeutics announced new three-year follow-up data from the continuing Phase III trial of LUXTURNA™, an investigational, potential one-time gene therapy for the treatment of patients with vision loss due to confirmed biallelic RPE65-mediated inherited retinal disease. [Spark Therapeutics, Inc.] Press Release

Alnylam Initiates ENVISION Phase III Clinical Study with Givosiran, an Investigational RNAi Therapeutic for the Treatment of Acute Hepatic Porphyrias (AHPs)

Alnylam Pharmaceuticals, Inc. announced the initiation of the ENVISION Phase III clinical study with givosiran, a subcutaneously administered, investigational RNAi therapeutic targeting aminolevulinic acid synthase 1 for the treatment of AHPs. [Alnylam Pharmaceuticals, Inc.] Press Release

Celsion Files Immunotherapy Clinical Protocol for the Evaluation of GEN-1 to Treat Newly Diagnosed Ovarian Cancer

Celsion Corporation announced the submission of its Phase I/II clinical trial protocol to the FDA for GEN-1, the company’s DNA-based immunotherapy for the localized treatment of ovarian cancer. [Celsion Corporation] Press Release

Cell and Gene Therapy Catapult Research Highlights the UK’s Growing Global Leadership in Innovative Cell and Gene Therapy Industry

With an increased focus from commercial ventures on pre-clinical research and clinical trials and a significant advance in cell and gene therapy manufacturing capability, this follows a consistent trend of growth across the industry since the Cell and Gene Therapy Catapult was established five years ago. [Cell and Gene Therapy Catapult] Press Release

Celgene Corporation and bluebird bio Announce bb2121 Anti-BCMA CAR-T Cell Therapy Has Been Granted Breakthrough Therapy Designation from FDA and PRIME Eligibility from EMA for Relapsed and Refractory Multiple Myeloma

Celgene Corporation and bluebird bio, Inc. announced that bb2121, a CAR-T therapy targeting b-cell maturation antigen (BCMA) in previously treated patients with multiple myeloma, has been granted Breakthrough Therapy Designation by the FDA and PRIority MEdicines (PRIME) eligibility by the European Medicines Agency (EMA). [Celgene Corporation] Press Release

Seattle Children’s Opens First CAR T-Cell Immunotherapy Trial in the U.S. for Children and Young Adults with Leukemia that Targets CD22 and CD19 Proteins Simultaneously

Seattle Children’s has opened the first CAR T-cell immunotherapy trial in the U.S. for children and young adults with relapsed or refractory CD19- and CD22-positive acute lymphoblastic leukemia that will simultaneously attack two targets on cancer cells. [Seattle Children’s Hospital] Press Release

Seattle Children’s Launches $1 Billion Fundraising Campaign to Transform Children’s Health

In order to expand Seattle Children’s T-cell immunotherapy research capabilities, a portion of the campaign will help fund Building Cure, a 540,000 square foot research facility in the heart of downtown Seattle. [Seattle Children’s Hospital] Press Release

Children’s Hospital Oakland Scientists Conduct First-Ever Gene Editing Inside a Human Body

A University of California, San Francisco Benioff Children’s Hospital Oakland’s adult patient underwent a landmark gene editing therapy – marking the first time that genome editing has been done inside a human body in an effort to change the DNA of a patient with a rare genetic disease. [University of California, San Francisco Benioff Children’s Hospital Oakland] Press Release | Editorial

Cancer Immunotherapy Company Tries to Explain Deaths in Recent Trial

More than a year after five leukemia patients died from an experimental treatment involving genetically engineered immune cells, its developer believes it has a better handle on what went wrong—and possibly how to prevent a repeat of the tragedy. [ScienceInsider] Editorial

ITUS Corporation Licenses CAR-T Technology from The Wistar Institute

ITUS Corporation announced that it has executed an exclusive worldwide license agreement with The Wistar Institute for a CAR-T technology aimed initially at treating ovarian cancer, and eventually additional solid tumors. [ITUS Corporation] Press Release

WuXi AppTec Selected by IQVIA Stem Cell Center to Serve as GMP Manufacturer of Advanced Therapies

WuXi AppTec announced that its Advanced Therapy Unit based in Philadelphia has been selected as a manufacturing partner for advanced therapies through the California Institute for Regenerative Medicine funded IQVIA Stem Cell Center. [WuXi AppTec (PR Newswire Association LLC.)] Press Release

UK Government Announces Research-Spending Hike ahead of Budget

The government said that it would boost public spending on research and development to £12.5 billion in 2021–22, an increase of £500 million on what is already planned for the year before. The rise builds on a surprise announcement made last year, when politicians promised yearly increases in research funding until 2020. [Nature News] Editorial

FDA Announces Comprehensive Regenerative Medicine Policy Framework

The FDA announced a comprehensive policy framework for the development and oversight of regenerative medicine products, including novel cellular therapies. The framework – outlined in a suite of four guidance documents – builds upon the FDA’s existing risk-based regulatory approach to more clearly describe what products are regulated as drugs, devices, and/or biological products. [U.S. Food and Drug Administration] Press Release

African Scientists Get Their Own Open-Access Publishing Platform

Africa’s academy of science has announced that it will launch an open-access publishing platform — the first of its kind aimed exclusively at scientists on the continent. [Nature News] Editorial

FDA

Authorizations of Emergency Use of In Vitro Diagnostic Devices for Detection of Zika Virus; Availability (FR Doc. No:2017-25010) Notice

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