Gene Editing for Immune Cell Therapies

Autologous T cells that have been genetically modified to express a chimeric antigen receptor targeting the B cell antigen CD19 have yielded remarkable clinical responses in patients with B cell malignancies, and are now on the market as anticancer ‘drugs’. [Nat BiotechnolAbstract

The Next Generation of CRISPR-Cas Technologies and Applications

New advances are considerably improving the understanding of biological processes and are propelling CRISPR-Cas-based tools towards clinical use in gene and cell therapies. [Nat Rev Mol Cell BiolAbstract

Principles of and Strategies for Germline Gene Therapy

The authors discuss novel strategies and provide a path forward for safe, high-efficiency germline gene therapy that may provide a promising new paradigm for preventing the passage of deleterious genes from parent to child. [Nat MedAbstract

Industry News

Amicus Therapeutics and the University of Pennsylvania Announce Major Expansion of Gene Therapy Collaboration

Amicus Therapeutics and the Perelman School of Medicine at the University of Pennsylvania announced a major expansion to their collaboration with rights to pursue collaborative research and development of novel gene therapies for lysosomal disorders and 12 additional rare diseases. [Amicus Therapeutics] Press Release

Rexgenero Successfully Expands Manufacturing of REX-001 through Partnership with German Red Cross Blood Donor Service

Rexgenero announced that it has successfully expanded manufacturing of its lead development candidate REX-001 through a partnership with the German Red Cross Blood Donor Service in Frankfurt, Germany, signed earlier this year. [Rexgenero] Press Release

Calidi Biotherapeutics Announces a Collaborative Study with the National Institutes Of Health (NIH) on the Therapeutic Potential of Oncolytic Viruses Delivered by Mesenchymal Stem Cells

Calidi Biotherapeutics, Inc. announced their ongoing scientific collaboration with David F. Stroncek, M.D., Chief, Cell Therapy Section at the Department of Transfusion Medicine, NIH to jointly study the impact of different stem cell carriers on the therapeutic potential of oncolytic viruses in the treatment of cancer. [Calidi Biotherapeutics, Inc. (PR Newswire Association LLC.)] Press Release

Orchard Therapeutics, Fondazione Telethon and Ospedale San Raffaele Announce Exclusive Worldwide License Agreement for the Treatment of MPS-I

Orchard Therapeutics, Fondazione Telethon and Ospedale San Raffaele announced that Orchard has been granted an exclusive worldwide license to intellectual property rights to research, develop, manufacture and commercialize the ex vivo autologous hematopoietic stem cell gene therapy program for the treatment of Mucopolysaccharidosis Type I (MPS-I) developed by the San Raffaele-Telethon Institute for Gene Therapy. [Orchard Therapeutics] Press Release

BioMarin Announces that Phase III Cohort of Valoctocogene Roxaparvovec, Gene Therapy Study in Severe Hemophilia A Met Pre-Specified Criteria for Regulatory Submissions in the US and Europe

BioMarin Pharmaceutical Inc. announced that its investigational gene therapy, valoctocogene roxaparvovec, for adults with severe hemophilia A achieved pre-specified clinical criteria for regulatory review in the US and Europe. [BioMarin Pharmaceutical Inc.]Press Release

Rejenevie™ Therapeutics Announces Start of Phase II Immune Restoration Treatment Clinical Trial

Rejenevie™ Therapeutics announced the commencement of the company’s Phase II clinical trial to study the safety and effectiveness of its proprietary AR-100 immune restoration therapy to boost the function of the aging immune system. [Rejenevie™ Therapeutics] Press Release

BrainStorm Announces Second Clinical Site to Enroll Patients in Its Progressive MS Phase II Study

BrainStorm Cell Therapeutics Inc. announced that the Stanford University School of Medicine will serve as the second contracted clinical study site for the company’s Phase II open-label, multicenter study of repeated intrathecal administration of autologous MSC-NTF cells in participants with progressive Multiple Sclerosis (MS). [BrainStorm Cell Therapeutics Inc.] Press Release

REGENXBIO Announces Completion of Dosing for Phase I/IIa Clinical Trial of RGX-314 in Wet AMD

REGENXBIO Inc. announced it completed dosing across all five cohorts in the Phase I/IIa clinical trial of RGX-314 for the treatment of wet age-related macular degeneration (wet AMD). [REGENXBIO Inc.] Press Release

BioMarin Provides Three Years of Clinical Data from Ongoing Phase I/II Study of Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A

BioMarin Pharmaceutical Inc. announced an update to its previously reported results of an open-label Phase I/II study of valoctocogene roxaparvovec, an investigational gene therapy treatment for adults with severe hemophilia A. [BioMarin Pharmaceutical Inc.]Press Release

NantKwest Launches First-in-Human Targeted NK Phase I Clinical Trial of CD19 T-haNK Cell Therapy in Lymphoma Patients

NantKwest Inc. announced that the company’s t-haNK investigational new drug application has cleared FDA review and the program has now transitioned to a first-in-human clinical trial targeting CD19 t-haNK in advanced B-cell lymphoma. [NantKwest Inc.]Press Release

bluebird bio Announces EU Conditional Marketing Authorization for ZYNTEGLO™ (Autologous CD34+ Cells Encoding βA-T87Q-globin Gene) Gene Therapy for Patients 12 Years and Older with Transfusion-Dependent β-Thalassemia Who Do Not Have β00Genotype

bluebird bio, Inc. announced that the European Commission has granted conditional marketing authorization for ZYNTEGLO™, a gene therapy for patients 12 years and older with transfusion-dependent β-thalassemia who do not have a β00 genotype, for whom hematopoietic stem cell (HSC) transplantation is appropriate but a human leukocyte antigen-matched related HSC donor is not available. [bluebird bio, Inc.] Press Release

Passage Bio Announces Third Gene Therapy Development Program in Krabbe Disease and Supports Million Dreams Fundraising Gala

Passage Bio announced that its third clinical trial program will be for infantile Krabbe disease, an inherited disease that causes progressive damage to the nervous system. [Passage Bio] Press Release

Ziopharm Oncology Announces Exclusive License with National Cancer Institute to Identify and Use T-Cell Receptors Targeting Neoantigens for Cancer with Sleeping Beauty Platform

Ziopharm Oncology, Inc. announced an exclusive licensing agreement with the National Cancer Institute for intellectual property for the development and commercialization of cell therapies for cancer. [Ziopharm Oncology, Inc.] Press Release

Fibrocell Receives FDA Regenerative Medicine Advanced Therapy Designation for FCX-007 Gene Therapy for the Treatment of RDEB

Fibrocell Science, Inc. announced that the FDA has granted the Regenerative Medicine Advanced Therapy designation to FCX-007, the company’s gene therapy candidate for the treatment of recessive dystrophic epidermolysis bullosa (RDEB)—a devastating, genetic skin disease with high mortality. [Fibrocell Science, Inc.] Press Release

Mesoblast Initiates Rolling Submission of Biologics License Application (BLA) to US FDA for Remestemcel-L in the Treatment of Acute Graft versus Host Disease

Mesoblast Limited announced that it has filed the first component of a rolling submission for a BLA to the FDA for remestemcel-L in the treatment of children with steroid-refractory acute graft versus host disease, a life-threatening complication of an allogeneic bone marrow transplant. [Mesoblast Limited] Press Release

Iovance Biotherapeutics Announces New Facility to Support US Production of Tumor Infiltrating Lymphocyte Cell Therapy Products

Iovance Biotherapeutics, Inc. announced that it has entered into a lease agreement to build an approximately 136,000 square foot commercial-scale production facility in Philadelphia for commercial and clinical production of autologous tumor-infiltrating lymphocyte products, including its candidate lifileucel. [Iovance Biotherapeutics, Inc.]Press Release

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