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A custom-made, one-dose gene therapy, Zynteglo is indicated for such patients who need red blood cells (RBCs) transfusions on a regular basis. The latest approval is based on findings from the Phase III clinical trials, HGB-207 (Northstar-2) and HGB-212 (Northstar-3), and the long-term follow-up LTF-303 study.
Myeloid Therapeutics has raised $73m to support the continued clinicaldevelopment of its lead cell therapy programme, MT-101, in Phase I/II trials for T cell lymphoma. MT-302 is a TROP2-targeting in vivo chimeric antigen receptor (CAR) that has been designed to express in the myeloid compartment.
Verve Therapeutics and Eli Lilly and Company have entered an exclusive research partnership to advance the former’s preclinical stage in vivogene editing programme targeting lipoprotein(a) (Lp(a)) to treat atherosclerotic cardiovascular disease (ASCVD). Blood concentrations of Lp(a) are determined almost entirely by inheritance.
Clinical-stage genome editing company Intellia Therapeutics has received clearance from the US Food and Drug Administration (FDA) for its Investigational New Drug (IND) application to start a pivotal phase III trial of NTLA-2001 for the treatment of transthyretin (ATTR) amyloidosis with cardiomyopathy.
Forbion European Acquisition has entered a definitive business combination agreement with biotechnology company enGene to create a combined biotechnology company to develop next-generation non-viral, locally administered gene therapies. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.
Merck (MSD outside North America) has entered a partnership agreement with Orna Therapeutics for discovering, developing and marketing various programmes based on next-generation RNA technology. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.
The two partners will take an in vivo approach to the problem of so-called sensorineural hearing loss (SNHL) looking at ways to replace sound-detecting hair cells in the inner ear (cochlea) that become damaged in this type of deafness. An estimated 1.57 Astellas is already working on other approaches to treating hearing loss.
LONDON–( BUSINESS WIRE )– Ixaka Ltd , an integrated cell and gene therapy company focused on the natural power of the body to cure disease, launches today. The new business will continue to develop Ixaka’s proprietary technologies – concentrated multi-cell therapies (MCTs) and targeted nanoparticle (TNP) therapeutics.
Vertex Pharma has ramped up its involvement in gene-editing medicines for the second time in a week, paying Obsidian Therapeutics $75 million upfront to access its technology platform. . Vertex is also offering up to $1.3 For example, adding a small-molecule might stabilise the medicine, allowing levels of the target protein to increase.
those that modify the expression of an individual’s genes or repair abnormal genes) has entered clinical practice, including 11 RNA therapeutics, 2 in vivogene therapies, and 2 gene-modified cell therapies. Here are 5 considerations when developing a communications strategy for a novel genetic medicine. #1.
“Our company and R&D portfolio are entering into an exciting phase, as evidenced by the recent close of Series B financing and submission of the first gene editing product IND in China,” said Dong Wei, Ph.D.?CEO Dr. Zhang has around 20 years of experience in research and drug development in both industry and academia in the US.
According to a recent review published in Nature , as of April 2022 almost 1,800 active cell therapy clinical trials were listed in ClinicalTrials.gov, a 33 percent increase from 2021. The rapidly expanding landscape of cell therapy trials has put an operational strain on many clinical sites due to limited resources and infrastructure. “In
Awareness of rare diseases is growing, and with a better understanding of the pathophysiology of many rare diseases, innovative treatment options are emerging, like gene therapies that can treat the root cause of rare genetic diseases and potentially provide long-term symptom relief, or even a definitive cure.
The Chinese biopharmaceutical industry is growing in leaps and bounds, but there is still a huge unmet need when it comes to getting patients access to the breakthrough therapeutic modalities and platforms like RNAi, cell and gene therapy and others. A solution could be at hand with Overland Pharmaceuticals.
The Company is required to develop and submit to the FDA an improved potency assay prior to enrolling the efficacy phase of the RUBY trial. EDIT-301 is an experimental, ex vivogene editing cell medicine in development for the treatment of sickle cell disease.
Bayer strengthens gene therapy portfolio with lipid nanoparticle technology from Acuitas Therapeutics Bayer AG is joining forces with Acuitas Therapeutics, Inc., a biotechnology company specializing in the development of lipid nanoparticle (LNP) delivery systems for molecular therapeutics.
The award honors McHutchison’s work in developing life-changing and curative therapies for patients with the hepatitis C virus. Axovant – Kristin Vuori was named to the board of directors at Axovant Gene Therapies Ltd. OncoSec Medical – Sanda Aung was named chief clinicaldevelopment officer for OncoSec Medical Incorporated.
The treatment induced HPV-specific T-cell responses and was associated with increased levels of immune cell infiltration in the tumors and expression of genes associated with activation of the immune system.
Key findings of the trial:
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Valentine On November 19, 2024, FDA released a draft guidance titled Frequently Asked Questions Developing Potential Cellular and Gene Therapy Products. academic to industry) involved in developing CGTs, the guidance starts by summarizing some of the fundamentals of opening an IND.
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