Cell Therapy News

Cell Therapy News is an online resource dedicated to sharing the latest cell therapy, gene therapy, and regenerative medicine research.

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Nobel Prizes Are Globally Unrepresentative — the Nomination Process Must Be Opened Up

October 10, 2024

The geography of science is changing. As a result, a big push is needed to broaden the pool of scientists eligible to nominate their...

AAV Gene Therapy for Duchenne Muscular Dystrophy: The EMBARK Phase III Randomized Trial

October 9, 2024

[Nature Medicine] A Phase III EMBARK study aimed to assess the efficacy and safety of delandistrogene moxeparvovec in patients with Duchenne muscular dystrophy (DMD) was conducted . Ambulatory males with DMD, ≥4 years to <8 years of age, were randomized and stratified by age group and North Star Ambulatory Assessment score to single-administration intravenous delandistrogene moxeparvovec or placebo.

Single-Cell Analysis of the Multiple Myeloma Microenvironment after Gamma-Secretase Inhibition and CAR T Cell Therapy

October 8, 2024

[Blood] Leveraging a Phase I clinical trial of the gamma-secretase inhibitor, crenigacestat, with anti-BCMA CAR T cells, researchers utilized single-nuclei RNA sequencing and Assay for Transposase-Accessible Chromatin sequencing to characterize the effects of gamma-secretase inhibitor on the tumor microenvironment.

Solid Cancer-Directed CAR T-Cell Therapy That Attacks Both Tumor and Immunosuppressive Cells via Targeting PD-L1

October 7, 2024

[Molecular Therapy Oncology] The authors developed a novel PD-L1 CAR using our humanized anti-PD-L1 monoclonal antibody, designed to simultaneously target tumor and immunosuppressive cells.

Point-of-Care Manufacturing of Anti-CD19 CAR-T Cells Using a Closed Semi-Automated Production Platform: Good and Bad Experiences from an Academic in Thailand

October 7, 2024

[Molecular Therapy] Investigators demonstrated the feasibility of the point-of-care manufacturing of anti-CD19 CAR-T cells from heavily pretreated patients and healthy graft donors at an academic medical center in Thailand using a closed semi-automated production platform, CliniMACS Prodigy, and established in-process quality control and release testing to ensure their identity, purity, sterility, safety, and potency.

Engineering Memory T Cells as a Platform for Long-Term Enzyme Replacement Therapy in Lysosomal Storage Disorders

October 7, 2024

[Molecular Therapy] The authors tested genetically engineered, alpha-L-iduronidase (IDUA)-expressing Tm as a cellular therapy in an immunodeficient mouse model of MPS I. Theirr esults demonstrated that a single dose of engineered Tm led to detectable IDUA enzyme levels in the blood for up to 22 weeks and reduced urinary GAG excretion.

Transplantation of Human Pluripotent Stem Cell-Derived Retinal Sheet in a Primate Model of Macular Hole

October 7, 2024

[Stem Cell Reports] Using a non-human primate model, investigators evaluated whether human embryonic stem cell-derived retinal organoid sheet transplantation can be an effective option for treating macular hole.

Medicine Nobel Awarded for Gene-Regulating ‘MicroRNAs’

October 7, 2024

[Nature News] The 2024 Nobel Prize in Physiology or Medicine has been awarded to two geneticists who discovered microRNAs, a class of tiny RNA molecules that help to control how genes are expressed in multicellular organisms.

World-First Therapy Using Donor Cells Sends Autoimmune Diseases into Remission

October 4, 2024

[Nature News] One woman and two men with severe autoimmune conditions have gone into remission after being treated with bioengineered and CRISPR-modified immune cells1. The three individuals from China are the first people with autoimmune disorders to be treated with engineered immune cells created from donor cells, rather than ones collected from their own bodies.

Mechanical Strategies to Promote Vascularization for Tissue Engineering and Regenerative Medicine

October 4, 2024

[Burns & Trauma] Scientists summarizes the influence and contribution of both scaffold-based and external stimulus-based mechanical strategies for vascularization in tissue engineering and elucidates the underlying mechanisms involved.

CD56-Targeted In Vivo Genetic Engineering of Natural Killer Cells Mediates Immunotherapy for Acute Myeloid Leukemia

October 4, 2024

[Nanoscale] In vivo CD56+ NK cell genetic engineering is hypothesized through the CD56-directed delivery of the pSMP-EzH2 shRNA plasmid encapsulated in chitosan nanoparticles.

Pre-Clinical Development of AP4B1 Gene Replacement Therapy for Hereditary Spastic Paraplegia Type 47

October 3, 2024

[EMBO Molecular Medicine] Investigators reported that a single delivery of adeno-associated virus serotype 9 expressing hAP4B1 (AAV9/hAP4B1) into the cisterna magna leads to widespread gene transfer and restoration of various hallmarks of disease, including AP-4 cargo mislocalisation, calbindin-positive spheroids in the deep cerebellar nuclei, anatomical brain defects and motor dysfunction, in an SPG47 mouse model.

Published since 2002, Cell Therapy News is our longest running online publication. Our editorial team curates the top publications, reviews, and news on in vivo gene therapy, cell therapy, and immunotherapy research. With over 15,000 subscribers and followers across our website, email newsletter, and Twitter feed, we are proud to facilitate the communication of cutting-edge cell therapy innovations.