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MONDAY, June 12, 2023 -- A clinical trial that’s attempting to discover a cure for sickle cell disease has found a new gene therapy to be safe and successful in four patients. Two of the patients were treated at Cleveland Clinic Children’s in.
Since a landmark paper in 2022, drugmakers have begun nearly a dozen trials of cell therapies for lupus, with more set to start. Here’s why their efforts are worth watching.
The offering comes days after the publication of a paper showing what analysts said was early proof that “off-the-shelf” cell therapies can treat inflammatory diseases.
For decades, scientists have dreamt of a future where genetic diseases, such as the blood clotting disorder hemophilia, could be a thing of the past. Gene therapy, the idea of fixing faulty genes with healthy ones, has held immense promise. But a major hurdle has been finding a safe and efficient way to deliver those genes.
1, 2023 -- A new gene therapy for sickle cell disease was deemed safe by a U.S. WEDNESDAY, Nov. Food and Drug Administration advisory panel on Tuesday, paving the way for full approval by early December. The FDA had already decided that the.
In an important step toward more effective gene therapies for brain diseases, researchers have engineered a gene-delivery vehicle that uses a human protein to efficiently cross the blood-brain barrier and deliver a disease-relevant gene to the brain in mice expressing the human protein.
A new gene therapy treatment for Duchenne muscular dystrophy (DMD) shows promise of not only arresting the decline of the muscles of those affected by this inherited genetic disease, but perhaps, in the future, repairing those muscles.
The company claims the results support exploring the possibility of an accelerated approval, citing flexibility by FDA officials in reviewing rare disease gene therapies.
TUESDAY, July 16, 2024 -- Hormone therapy for breast cancer might reduce a woman’s later risk of dementia and Alzheimer’s disease, a new study finds.Overall, hormone therapy is associated with a 7% lower risk of developing Alzheimer’s or a related d.
Food and Drug Administration on Friday approved two milestone gene therapies for sickle cell disease, including the first treatment ever approved that uses gene-editing technology. FRIDAY, Dec. 8, 2023 -- The U.S. Casgevy, developed by Vertex.
Two-year-old Kate Therapeutics is launching publicly with $51 million in funding and a licensing deal with Astellas Pharma for a neuromuscular disease gene therapy.
The pharma plans for the new Berkeley facility to produce clinical, and potentially commercial, supplies of a Parkinson’s disease cell therapy being tested by its subsidiary BlueRock.
TUESDAY, June 18, 2024 -- There's more good news in the battle against sickle cell disease, with another trial finding CRISPR gene-editing therapy delivering impressive results for patients.“It’s It’s encouraging that this gene-editing treatment con.
Promising preclinical results show hematopoietic stem cell therapy was effective in rescuing memory loss, neuroinflammation and beta amyloid build-up in a mouse model of Alzheimer's disease.
By inserting an entire gene, the tool offers a one-and-done approach that overcomes hurdles from CRISPR gene editing technology -- which is programmed to correct individual mutations -- offering a promising step forward for gene therapy.
Scientists have made a `paradigm shifting' discovery on the mechanisms required for learning and memory that could lead to new therapies for Alzheimer's disease and potentially Down syndrome.
Like many of its cell therapy peers, Artiva, which originally sought an IPO in 2021, has shifted its strategy in hopes of riding a recent wave of investor interest in autoimmune disease research.
Phase 2 results in inflammatory bowel disease hinted the therapy could be more potent than similar medicines from Merck and Roche, each of which were acquired in multibillion-dollar deals.
” But reports of relapses in some patients drew questions about the therapies’ ultimate potential. One expert described trial results presented at EULAR last week as “unprecedented.”
31, 2023 -- A type of gene therapy that precisely "edits" a key bit of DNA might offer a new way to treat sickle cell disease -- a painful inherited condition that largely strikes Black children and adults. THURSDAY, Aug. That's according to a new.
29, 2023 -- A new cellular therapy improved learning and memory in mice with Alzheimer's disease, researchers report.The therapy -- developed at the University of Nebraska Medical Center (UNMC) -- relies on both the immune system to. FRIDAY, Dec.
One day, researchers want to use switches of this kind to trigger cell therapies for various metabolic diseases. Researchers have developed a new gene switch that can be activated using a commercially available nitroglycerine patch applied to the skin.
Food and Drug Administration on Friday approved two milestone gene therapies for sickle cell disease, including the first treatment ever approved that uses gene-editing technology.Casgevy, developed by Vertex. FRIDAY, Dec. 8, 2023 -- The U.S.
clearance of Lenmeldy, for a rare and inherited metabolic disease, triggers an additional payout related to Kyowa Kirin’s recent deal to acquire the once high-flying gene therapy developer.
The company claimed Phase 2 study results prove the therapy’s promise treating myasthenia gravis. But shares lost more than a quarter of their value amid questions about the data.
This discovery opens up new opportunities for therapeutic intervention to control cholesterol uptake that could complement other therapies and potentially save lives. Researchers have discovered the mechanism by which cholesterol in our diet is absorbed into our cells.
Seven young boys given Bluebird's Skysona later developed blood cancers, findings that could shape how doctors balance the gene therapy’s risks against its benefit.
The full results spotlight cell therapy’s potential to treat lupus and other inflammatory conditions, but also the problems drug developers must solve first.
28, 2023 -- Stem cells injected into the brains of multiple sclerosis patients appear to protect them against further damage from the degenerative disease, a new study shows.MS TUESDAY, Nov. occurs when the body’s own immune system attacks and.
Study results disclosed at the American Society of Hematology meeting Saturday suggested potential for cell therapy in lupus and pointed out drug trial limitations.
A large-scale clinical trial of treatment strategies for Crohn's disease has shown that offering early advanced therapy to all patients straight after diagnosis can drastically improve outcomes, including by reducing the number of people requiring urgent abdominal surgery for treatment of their disease by ten-fold.
Fresh off a Series C fundraise, Kriya has acquired the privately held Tramotane Therapeutics and its preclinical candidate for the widely prevalent liver disease.
The figure depicts the neuroprotective role of exendin-4 in Alzheimer's disease and Parkinson's disease. Due to the complex pathological processes underlying neurodegeneration, at present, there is no viable therapy available for neurodegenerative disorders.
The research could revolutionize therapies for complex conditions like autoimmune disease and cancer. Bioengineers have developed a new construction kit for building custom sense-and-respond circuits in human cells.
The gene editing biotech is shelving two of its most advanced cancer drugs, and joining a growing group of companies exploring cell-based medicines for inflammatory diseases like lupus.
Such neurons could be used to treat spinal cord injuries or diseases such as ALS. Researchers devised a process to convert a skin cell directly into a neuron, eliminating the need to generate induced pluripotent stem cells.
The new T cell atlas is publicly available and should help in the development of new drug therapies for immune-mediated diseases. The discoveries were made possible by a newly developed technology they call ReapTEC.
In buying Westlake-backed Kate Therapeutics, Novartis gets a handful of preclinical gene therapies targeting diseases like Duchenne muscular dystrophy and myotonic dystrophy type 1.
A new sickle cell diseasetherapy developed by CRISPR Therapeutics and Vertex Pharmaceuticals is now approved in the U.S. This is the story of how it came to be.
The company’s haul is the latest sign brain drugs are recapturing the interest of investors after years of being passed over for other areas of research.
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