Cell Therapy News Volume 19.38 | Nov 19 2018

TOP STORY

RNAi Modulation of Placental sFLT1 for the Treatment of Preeclampsia

Scientists identified short interfering RNAs that selectively silence the three soluble vascular endothelial growth factor receptor FLT1 (sFLT1) mRNA isoforms primarily responsible for placental overexpression of sFLT1 without reducing levels of full-length FLT1 mRNA. [Nat Biotechnol] Abstract

(Ranked by impact factor of the journal)

In Vivo Hematopoietic Stem Cell Gene Therapy Ameliorates Murine Thalassemia Intermedia

Investigators developed a simple in vivo hematopoietic stem/progenitor cell (HSPC) gene therapy approach that involved HSPC mobilization and an intravenous injection of integrating HDAd5/35⁺⁺ vectors. [J Clin Invest] Abstract

AAV Gene Transfer with Tandem Promoter Design Prevents Anti-Transgene Immunity and Provides Persistent Efficacy in Neonate Pompe Disease Mice

Scientists showed that the use of efficient tandem promoters prevents immune responses to acid-alpha-glucosidase (GAA) following systemic AAV gene transfer in immunocompetent Gaa^-/- mice [Mol Ther Methods Clin Dev] Abstract | Graphical Abstract | Full Article

AAV-Mediated Progranulin Delivery to a Mouse Model of Progranulin Deficiency Causes T Cell-Mediated Toxicity

Researchers used AAV9 to deliver progranulin gene (GRN) to the lateral ventricle to achieve widespread expression in the Grn null mouse brain. They found that despite a global increase in progranulin, overexpression resulted in dramatic and selective hippocampal toxicity and degeneration affecting neurons and glia. [Mol Ther] Abstract | Graphical Abstract

Mesenchymal Stem Cells and Induced Bone Marrow-Derived Macrophages Synergistically Improve Liver Fibrosis in Mice

The authors describe a novel therapeutic approach for cirrhosis using mesenchymal stem cells and colony-stimulating factor-1-induced bone marrow-derived macrophages and analyzed the mechanisms underlying fibrosis improvement and regeneration. [Stem Cells Transl Med] Full Article

Safe and Efficient Novel Approach for Non-Invasive Gene Electrotransfer to Skin

Researchers aimed to increase gene electrotransfer (GET) efficacy in skin, by optimizing electric pulse parameters and the design of electrodes. They evaluated the safety of the novel approach by assaying the thermal stress effect of GET conditions and the biodistribution of a cytokine expressing plasmid. [Sci Rep] Full Article

Resistin Promotes Cardiac Homing of Mesenchymal Stem Cells and Functional Recovery after Myocardial Ischemia/Reperfusion via the ERK1/2-MMP-9 Pathway

Both immunostaining and flow cytometric studies showed that resistin treatment promoted adipose-derived mesenchymal stem cells (ADSC) myocardial homing three days after intravenous injection. Echocardiographic studies showed that ADSC-resistin, but not ADSC-vehicle, significantly improved the left ventricular ejection fraction. [Am J Physiol Heart Circ Physiol] Abstract

Gene Therapy by Lentivirus-Mediated RNA Interference Targeting Extracellular-Regulated Kinase Alleviates Neuropathic Pain In Vivo

Researchers investigated the effects of extracellular-regulated kinase knockdown via lentivirus-mediated RNA interference on allodynia in rats with chronic compression of the dorsal root ganglia and uncovered the potential mechanisms. [J Cell Biochem] Abstract

Efficiency of Dual siRNA Mediated Gene Therapy for Intervertebral Disc Degeneration (IVDD)

Scientists demonstrated the regenerative potential of small interfering RNA (siRNA) designed against caspase 3 and ADAMTS5 genes in an in vitro and animal model of disc degeneration. [Spine J] Abstract

Intratumoral IFN-α Gene Delivery Reduces Tumor-Infiltrating Regulatory T Cells through the Downregulation of Tumor CCL17 Expression

Investigators examined whether IFN-α affects the trafficking of regulatory T cells to the tumor. Since CT26 cells expressed CCL17 among Treg-attracting chemokines, they focused on its role in IFN-α-mediated Treg suppression. [Cancer Gene Ther] Abstract

AAV-Mediated Follistatin Gene Therapy Improves Functional Outcomes in the TIC-DUX4 Mouse Model of FSHD

To demonstrate the utility of TIC-DUX4 mice for therapeutic development, researchers tested a gene therapy approach aimed at improving muscle strength in DUX4-expressing muscles using adeno-associated virus (AAV) serotype 1.Follistatin, a natural myostatin antagonist. [JCI Insight] Full Article

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How to Design Preclinical Studies in Nanomedicine and Cell Therapy to Maximize the Prospects of Clinical Translation

The authors discuss special manifestations of problems in nanomedicine and in cell therapy, and describe mitigating strategies. Progress on reducing bias and enhancing reproducibility early on ought to enhance the translational potential of biomedical findings and technologies. [Nat Biomed Eng] Abstract

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

Ziopharm Oncology Provides Controlled IL-12 Update with Positive Data from Phase I Trial Presented

Ziopharm Oncology, Inc. provided an update on its controlled IL-12 platform following a presentation of positive data from its Phase I clinical trial in recurrent glioblastoma. [Press release from Ziopharm Oncology, Inc. discussing research presented at the 23^rd Annual Scientific Meeting and Education Day of the Society for Neuro-Oncology (SNO), New Orleans] Press Release

RXi Pharmaceuticals to Present New Data Demonstrating the Ability of sd-rxRNA to Enhance NK Cell Activity for Adoptive Cell Transfer

RXi Pharmaceuticals Corporation will present a poster highlighting data demonstrating the potential of self-delivering RNAi (sd-rxRNA) to improve NK cell potency in adoptive cell transfer. [Press release from RXi Pharmaceuticals Corporation discussing research presented at the 33^rd Annual Meeting & Pre-Conference Programs of the Society for Immunotherapy of Cancer (SITC), Washington] Press Release

Gamma-Delta (γδ) T Cell Therapy Combined with Checkpoint Inhibition Shows Potential Synergies In Vitro for Treatment of Glioblastoma

Incysus Therapeutics, Inc. announced that promising data from a pilot in vitro study combining checkpoint inhibitors with γδ T cell therapy will be presented. [Press release from Incysus Therapeutics, Inc. (Globe Newswire, Inc.) discussing research presented at the 23^rd Annual Scientific Meeting and Education Day of the Society for Neuro-Oncology (SNO), New Orleans] Press Release

Positive 12-Month Data on BioCardia’s Investigational CardiAMP Cell Therapy System for Heart Failure Presented

BioCardia^®, Inc. announced positive 12 month data for the roll-in cohort of its pivotal CardiAMP™ Heart Failure Trial studying the investigational CardiAMP Cell Therapy System in adult patients experiencing heart failure following a heart attack. [Press release from BioCardia^®, Inc. discussing research presented at the American Heart Association Scientific Sessions 2018, Chicago] Press Release

Celyad Presents Update on CYAD-01 Solid Tumor Clinical Program

Celyad announced updated clinical for the CYAD-01 program in solid tumors as well as translational research data. [Press release from Celyad discussing research presented at the 33^rd Annual Meeting & Pre-Conference Programs of the Society for Immunotherapy of Cancer (SITC), Washington] Press Release

BioTime and Asterias Biotherapeutics Enter Into Definitive Merger Agreement to Create Leading Cell Therapy Company

BioTime, Inc. and Asterias Biotherapeutics, Inc. announced that they have entered into a definitive merger agreement whereby BioTime will acquire all of the remaining outstanding common stock of Asterias that are not currently owned by BioTime. [BioTime, Inc.] Press Release

Phoenix Nest Inc. Licenses a Sanfilippo Syndrome Gene Therapy from the University of Manchester and Is Preparing for a Clinical Trial

Phoenix Nest Inc. exclusively licensed a gene therapy for Sanfilippo syndrome from The University of Manchester through its IP commercialization arm UMI3 Ltd. [Phoenix Nest Inc. (PR Newswire Association LLC.)] Press Release

Cabaletta Bio Emerges Out of University of Pennsylvania to Develop CAAR T Cell Technology to Treat B Cell-Mediated Autoimmune Diseases

Cabaletta Bio Inc. has signed an exclusive license agreement and executed two multi-year sponsored research agreements with the University of Pennsylvania for the discovery and development of engineered T cell therapy products for B cell-mediated autoimmune disease. [Cabaletta Bio Inc. (Globe Newswire Inc.)] Press Release

Myonexus Therapeutics Announces the Initiation of a Trial for the First-Ever Gene Therapy for Betasarcoglycanopathy, Also Known as Limb-Girdle Muscular Dystrophy Type 2E

Myonexus Therapeutics in collaboration with Nationwide Children’s Hospital’s Center for Gene Therapy and Sarepta Therapeutics, announced the initiation of a clinical trial evaluating MYO-101, a novel gene therapy candidate for beta-sarcoglycanopathy, also known as LGMD2E. [Myonexus Therapeutics] Press Release

Capricor to Meet with FDA under Its RMAT Designation to Discuss HOPE-2 Clinical Trial

Capricor Therapeutics announced that it will meet with the FDA to discuss clinical trial design, surrogate or intermediate endpoints and manufacturing processes for CAP-1002, Capricor’s novel cell therapy. [Capricor Therapeutics] Press Release

HemaCare Provides Starting Material for Three FDA Approved Cellular Therapies

HemaCare Corporation announced their involvement as a provider of leukapheresis process development material for 100% of the current FDA-approved immunocellular therapies, Kymriah^®, Yescarta^®, and Provenge^®. [HemaCare] Press Release

uniQure Announces Initial Topline Data from Dose-Confirmation Study of AMT-061 in Patients with Hemophilia B

uniQure N.V. announced initial clinical data in patients treated in the company’s Phase IIb dose-confirmation study of AMT-061, an investigational AAV5-based gene therapy containing a patent-protected FIX-Padua variant, for the treatment of patients with severe and moderately severe hemophilia B. [uniQure N.V.] Press Release

Promising Approach to Curing Spina Bifida Gets $5.6 Million from Stem Cell Agency

A new approach to repairing the defect that causes spina bifida was awarded $5.66 million by the Board of the California Institute for Regenerative Medicine. [California Institute for Regenerative Medicine] Press Release

4D Molecular Therapeutics Announces FDA Orphan Drug Designation Granted to 4D-110 for the Treatment of Choroideremia

4D Molecular Therapeutics announced that the FDA has granted orphan drug designation to 4D-110 for the treatment of choroideremia. [4D Molecular Therapeutics] Press Release

Rocket Pharmaceuticals Announces Clearance of IND for RP-L201 Gene Therapy for Leukocyte Adhesion Deficiency-I (LAD-I)

Rocket Pharmaceuticals, Inc. announced the clearance of the company’s IND application by the FDA for RP-L201. [Rocket Pharmaceuticals, Inc.] Press Release

SpinalCyte Announces Sustained 12-Month MRI Outcomes after CybroCell™ Injection

SpinalCyte, LLC announced that a single injection of modified human dermal fibroblasts, resulted in significant improvements of disc height and pain reduction 12 months after injection of the cell therapy for patients with degenerative disc disease. [SpinalCyte, LLC] Press Release

FDA Grants Orphan Drug Designation to RGX-181 Gene Therapy for the Treatment of CLN2 Form of Batten Disease

REGENXBIO Inc. announced the FDA granted Orphan Drug Designation to RGX-181, a one-time treatment candidate for late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease, one of the most common forms of Batten disease caused by mutations in the tripeptidyl peptidase 1 gene. [REGENXBIO Inc.] Press Release

Hemostemix Announces Development of an ACP-01 Allogeneic Process and Initiation of R&D for Autologous NCP-01 Neural Cellular Precursors

Hemostemix Inc. announced it has made additional findings surrounding its overall technology platform and initiation of R&D work on NCP-01, another one of its product pipeline candidates. [Hemostemix Inc.] Press Release

Adverum Announces First Patient Dosed in OPTIC Phase I Trial of ADVM-022 Gene Therapy in Wet Age-Related Macular Degeneration

Adverum Biotechnologies, Inc. announced that the first patient was dosed in the OPTIC Phase I trial evaluating ADVM-022 for patients with wet age-related macular degeneration. [Adverum Biotechnologies, Inc.] Press Release

Can Novartis Charge $4 Million for a One-Time Drug?

Novartis believes its new gene therapy is worth more than $4 million for a one-time dose, and the company has some data to back that up. But, with a global spotlight on the escalating cost of medicine, is it politically viable to set a new record for the world’s most expensive drug? [STAT News] Editorial

Duke University to Settle Case Alleging Researchers Used Fraudulent Data to Win Millions in Grants

Duke University in Durham, North Carolina, is on the verge of settling a case brought by a former employee who claims the university included faked data in applications and reports for federal grants worth nearly $200 million. [ScienceInsider] Editorial

Cancer Drug’s Stumbles Prompt Calls to Rethink How Immune Therapies Are Tested

The stunning failure of a once-promising cancer drug has got some researchers arguing that the field has moved too fast in its embrace of therapies that unleash the immune system. [Nature News] Editorial

French Science Behemoth Launches Research-Integrity Office

France’s national research centre, the CNRS, has announced plans to create its first office of research integrity to investigate scientific misconduct and promote good research practice. [Nature News] Editorial

Research Groups Attend HHS ‘Listening Session’ on Fetal Tissue Research amid Funding Fears

Science advocates who attended a “listening session” on the use of fetal tissue in medical research held by senior officials at the U.S. Department of Health and Human Services (HHS) in Washington, D.C., say they are optimistic that they were listened to and heard. But many researchers remain concerned about reports that President Donald Trump’s administration is considering withdrawing funding for such studies, which are fiercely opposed by antiabortion advocates. [ScienceInsider] Editorial

In Reversal, NSF Lifts Proposal Limits on Biologists

In a reversal, the National Science Foundation (NSF) will no longer restrict researchers to only one proposal submission per year to the biology directorate’s three core tracks in which they are listed as a principal investigator (PI) or co-PI. [ScienceInsider] Editorial

NIH

Prospective Grant of an Exclusive Patent License: Development and Commercialization of Chimeric Antigen Receptor (CAR) Therapies for the Treatment of FMS-Like Tyrosine Kinase 3 (FLT3) Expressing Cancers (FR Doc. No:2018-25197) Notice

TGA

Communication to Stakeholders: Autologous Human Cells and Tissues (HCT) Products Notice

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