This week: Cartherics (Melbourne, Australia) has entered agreements with two Australian universities to advance cellular therapies for cardiovascular disease, the US Food and Drug Administration (FDA; MD, USA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to a CAR T-cell therapy candidate for recurrent anaplastic thyroid cancer, and Oxford Nanopore Technologies (Oxford, UK) has announced a collaboration with Plasmidsaurus (CA, USA) to advance plasmid sequencing and develop new technologies for applications like gene therapy. The news highlights: Advancing cardiac cell ..read more

A recent study has uncovered how mechanisms in the kidney, activated by low salt and body fluid, could offer a new avenue for kidney regeneration. Researchers from the Keck School of Medicine of the University of Southern California (CA, USA) have explored the evolutionary and physiological adaptive responses of the kidney to improve our understanding of endogenous kidney tissue repair. They identify potential therapeutic targets that may aid the development of mechanism-based regenerative therapies for kidney disease regression. The kidney is one of several organs in the human body with limi ..read more

Tuesday 27 August 2024 09:00 [PDT]  12:00 [EDT]  17:00 [BST] Alternative time zones Cell and gene therapy manufacturers face challenges to achieving quality, consistency, and safety in the final product, mandating risk-based management of ancillary materials to help meet those goals. Safety, quality, consistency and effectiveness are defining features of cell and gene therapy therapeutic products. Achieving these features requires controlled and reproducible manufacturing workflows supported by rigorous, traceable documentation and consistent high-quality ancillary materials ..read more

This week: The US Food and Drug Administration (FDA; MD, USA) grants Priority Review to Atara Biotherapeutics’ (CA, USA) Biologics License Application (BLA) for tabelecleucel (tab-cel®), Vertex Pharmaceuticals (MA, USA) licenses Orum Therapeutics’ (Daejeon, South Korea) technology to develop novel conditioning agents and AffyImmune (MA, USA) appoints a new President and CEO. The news highlights: FDA grants Priority Review to Atara’s T-cell therapy for post-transplant lymphoproliferative disease Vertex licenses Orum’s technology to develop novel conditioning agents AffyImmune appoints new Pre ..read more

A large-scale study has found that women can get pregnant and give birth following allogeneic hematopoietic cell transplantation, refuting previous widespread claims that it was virtually impossible. Allogeneic hematopoietic cell transplantation (alloHCT) involves the transplantation of donor stem cells into patients. It is used to treat a number of blood disorders, including sickle cell anemia and leukemia. Due to continual improvements in procedures for alloHCT and supportive therapies, we are seeing growing numbers of long-term survivors who are keen to cultivate a healthy and socially nor ..read more

This week: A study detailed findings of a potential target for T-cell therapy in acute myeloid leukemia (AML), US$170 million was raised in Series B funding to advance research in retinal gene therapy. Plus, the Australian Therapeutic Goods Administration (TGA; Canberra, Australia) has given the green light for an in vivo chimeric antigen receptor (CAR) therapy for B-cell malignancies to be assessed in clinical trials. The news highlights: Data demonstrates a potential target for acute myeloid leukemia T-cell therapy US$170 million in Series B funding raised to propel eye gene therapy resear ..read more

A recent study has leveraged nanoparticle drug delivery to directly administer treatment to the site of injured tendons. Researchers from the University of Rochester (NY, USA) and University of Oregon (OR, USA ) have collaborated on a recent study combining their expertise in tendon cell biology and drug delivery systems to investigate a promising new approach to improve tendon healing. By reducing scar tissue formation using a targeted nanoparticle-based drug delivery system approach, this innovation aims to enhance regenerative, rather than fibrous, healing that naturally occurs in injured ..read more

A recent study has shown that stem cell technology could offer a viable treatment for Hirschsprung disease, potentially reducing the need for multiple surgeries. Hirschsprung disease is a rare genetic disorder occurring in around 1 in every 5,000 live births. Patients are born with some nerve cells missing in a portion of the large intestine, meaning it can’t contract normally to move stool. This can cause chronic constipation and colon swelling, leaving patients prone to painful enterocolitis and at risk of bowel perforation. The condition is generally diagnosed early and patients undergo su ..read more

This week: Clinical trials for a T-cell receptor-engineered T (TCR T) cell therapy for human papillomavirus (HPV)-associated solid tumors will commence following Investigational New Drug approval, the US Food and Drug Administration (FDA; MD, USA) has granted Priority Review for a bioengineered vascular replacement and approval of the Biologics License Application (BLA) for the gene therapy KRESLADI has been delayed. The news highlights: Clinical trials to commence for HPV-specific TCR T-cell therapy Priority Review for bioengineered vascular replacement Biologics License Application delayed ..read more

Researchers have utilized intestinal epithelial organoids to investigate how DNA modifications in gut cells may influence the risk of Crohn’s disease. In a recent study, researchers at the University of Cambridge (CUH; UK) leveraged patient-derived intestinal stem cells to grow intestinal epithelial organoids, providing a better understanding of Crohn’s disease (CD). The team performed genome-wide molecular profiling and functional analysis of these organoids, aiming to uncover potential mechanisms involved in the pathogenesis of CD. By using organoids to model CD instead of mice models, they ..read more