Scientists at University of Cambridge have used gene therapy to regenerate damaged nerve fibres in the eye, in a discovery that could aid the development of new treatments for glaucoma, one of the leading causes of blindness worldwide. Axons – nerve fibres – in the adult central nervous system (CNS) do not normally regenerate after
B cell acute lymphoblastic leukemia (B-ALL) blasts hijack the bone marrow (BM) microenvironment to form chemoprotective leukemic BM “niches,” facilitating chemoresistance and, ultimately, disease relapse. However, the ability to dissect these evolving, heterogeneous interactions among distinct B-ALL subtypes and their varying BM niches is limited with current in vivo methods. In this paper , New
Phase 1 Clinical Study will Evaluate ADI-001 Safety and Efficacy in Patients with B-cell non-Hodgkin’s lymphoma Adicet Bio, Inc., a biotechnology company discovering and developing allogeneic gamma delta T cell therapies for cancer and other diseases, today announced that the U.S. Food and Drug Administration (FDA) has cleared the Company’s Investigational New Drug (IND) application for ADI-001,
NIH-funded therapy will now be tested in humans A newly developed light-sensing protein called the MCO1 opsin restores vision in blind mice when attached to retina bipolar cells using gene therapy. The National Eye Institute, part of the National Institutes of Health, provided a Small Business Innovation Research grant to Nanoscope, LLC for development of
Scientists at the Centro Nacional de Investigaciones Cardiovasculares (CNIC) have discovered that neutrophils, the most abundant cells of the innate immune system, have many more functions in the body than previously thought. This finding suggests possible new treatments for many diseases, including cancer. In a study published in the journal Cell, the research team demonstrates that
In the past three decades the field of gene therapy has made remarkable progress surging from mere laboratory experiments to FDA-approved products which bring significant reduction in disease burden to patients who previously had no therapeutic options for their serious conditions. In this paper it’s reviewed the evolution of the gene therapy clinical research landscape
The RESTORE position paper represents the cumulative efforts of the RESTORE community, and the steering committee since the start of the RESTORE initiative in 2019. It outlines, in depth, the RESTORE view on why Europe should invest in Advanced Therapies, the roadblocks in Advanced Therapy development and implementation into patient care, and makes recommendations about
Chimeric antigen receptor (CAR) technology and its application to regulatory T cells (Tregs) has garnered interest among researchers in the field of cell and gene therapy. Merging the benefits of CAR technology with Tregs offers a novel and promising therapeutic option for durable reshaping of undesired immune responses following solid organ or hematopoietic stem cell
Despite the first clinical studies reporting on a correlation betweenthe migration potential of transplanted stem cells and their therapeutic efficacy, it is unclear if effective stem cell therapies requirereliable and sufficient delivery of the cells to the desired anatomiclocations, as well as survival of the cellular graft. Moreover, the many complex processes involved in stem
In the early days of COVID-19, the Alliance for Regenerative Medicine (ARM) was unsure how the pandemic and its accompanying economic downturn would affect the cell and gene therapy space. “It was a really specific time when the world and the markets were clearly reeling from the first appreciation for the seriousness of COVID-19,” Janet
