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Results from a phase 1/2 clinical trial of a novel stem cell treatment for cornea injuries found 14 patients treated and tracked for 18 months had a more than 90% success rate at restoring the cornea's surface and improvements in vision.
The vision of people with a rare inherited condition that causes them to lose much of their sight early in childhood was 100 times better after they received gene therapy to address the genetic mutation causing it.
Peter Marks’ decision to override the objections of agency staff and broaden use of Elevidys could have a “lasting impact” on gene therapy as well as the FDA, one analyst wrote.
Far fewer venture funding rounds were closed by cell and gene therapy developers over the first six months of 2024 than in prior years. Experts say there are several factors.
The agency's openness to a targeted pivotal study shows it’s still willing to consider accelerated clearance for Duchenne gene therapies despite questions about their effectiveness.
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.
WEDNESDAY, May 15, 2024 -- An experimental gene therapy could one day provide a first-ever cure for genital and oral herpes, researchers report.The gene therapy removed 90% or more of oral herpes infection in lab mice, and it also suppressed how.
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.
Gene therapy, the idea of fixing faulty genes with healthy ones, has held immense promise. 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. But a major hurdle has been finding a safe and efficient way to deliver those genes.
MONDAY, May 6, 2024 -- A new combination therapy appears to boost the response rate for liver cancer patients receiving immunotherapy, according to results from a phase 2 clinical trial.The combo therapy involves bavituximab, a drug that.
For years, therapy dogs have ruled the world of animal-assisted services (AAS), offering stress relief to college students, hospital patients, and those in need of emotional support.
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.
7, 2024 -- Light therapy: It's long been a go-to therapy for seasonal affective disorder (SAD), a kind of depression that can beset some people when winter looms and days shorten.But new research is suggesting that time spent in front. MONDAY, Oct.
Five years after helping launch Allogene Therapeutics, Pfizer is backing another developer of “off-the-shelf” cell therapies in a deal that gives the drugmaker the chance to acquire a multiple myeloma treatment.
The Food and Drug Administration approval of Beqvez comes as other gene therapies for the bleeding condition that were approved earlier struggle to gain traction.
MONDAY, May 6, 2024 -- An injectable gene therapy caused measurable improvements in vision among a small group of people with inherited blindness, an early-stage clinical trial says.Researchers recruited 14 people with Leber Congenital Amaurosis.
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 partnership between cell therapy delivery specialist Portal Biotechnologies and precision robotics maker Multiply Labs could address manufacturing hurdles, the CEOs said.
Regenerative heart therapies involve transplanting cardiac muscle cells into damaged areas of the heart to recover lost function. However, the risk of arrhythmias following this procedure is reportedly high.
WEDNESDAY, March 19, 2025 -- A patient has died while receiving Elevidys, a gene therapy for Duchenne muscular dystrophy, marking the first known death linked to the treatment. The company behind the drug, Sarepta Therapeutics, said that the.
This study marks an important step toward regenerative therapies to prevent heart failure. In mice, this protein was able to restore the heart by activating dormant repair genes without causing side effects, such as heart enlargement.
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.
MONDAY, Jan 6, 2025 -- Turns out, starting physical therapy earlier after a concussion may significantly improve recovery outcomes.A new study published recently in the Physical Therapy & Rehabilitation Journal followed just over 200 adults.
22, 2024 -- Light therapy could be a useful treatment for the most common form of age-related macular degeneration, a new study says.The therapy, called photobiomodulation or “red light” therapy, can reduce the risk of vision loss and. TUESDAY, Oct.
The company claims the results support exploring the possibility of an accelerated approval, citing flexibility by FDA officials in reviewing rare disease gene therapies.
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.
WEDNESDAY, May 1, 2024 -- Hormone replacement therapy can safely ease middle-aged women’s symptoms during early menopause, data from a major women’s health study show. Women younger than 60 can use hormone replacement to treat symptoms.
Elevidys sales have increased since the FDA made a controversial choice to expand the therapy's use. Now, Sarepta is abandoning a successor to its drug Exondys 51, citing an “evolving" treatment landscape.
Adults with hemophilia B saw their number of bleeding episodes drop by an average of 71 percent after a single infusion of gene therapy, according to the new results of an international Phase III clinical trial.
A new clinical trial of five children with inherited deafness found administering gene therapy in both ears led to restored hearing and speech, and additional gains including sound source localization, ability to hear in noisy environments, and for two children, abillity to appreciate music.
25, 2024 -- A new gene therapy approved earlier this year can serve as a sustainable single-dose treatment for people with hemophilia B, newly published clinical trial data shows. THURSDAY, Sept. People with hemophilia B saw their bleeding episodes.
THURSDAY, June 13, 2024 -- CAR-T cell therapy to treat blood cancers is safer than previously thought, with little risk that the immunotherapy will create secondary cancers, a new study finds.The U.S. Food and Drug Administration issued a warning.
Casgevy, the first CRISPR therapy approved by the FDA, will cost $2.2 million, while a competing genetic medicine also cleared Friday is priced at $3.1
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.
The negative results from the Phase 3 study, called Ciffreo, come a little more than a month after Pfizer said a patient died in another trial of the gene therapy.
A young man treated with Elevidys died of acute liver failure. The case may give doctors pause before prescribing the treatment in the future, some analysts wrote.
6, 2024 -- Gene therapy may restore vision to children and adults robbed of their sight by a rare inherited condition called Leber congenital amaurosis, researchers report.The illness is caused by mutations in the GUCY2D gene, which. FRIDAY, Sept.
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.
Food and Drug Administration has told drugmakers to add a boxed warning to a type of cancer treatment called CAR-T therapy, saying the treatment itself may sometimes cause a secondary. WEDNESDAY, Jan. 24, 2024 (Healthday News) -- The U.S.
22, 2024 -- Four years of hormone replacement therapy to help women deal with menopausal symptoms causes no harm to the brain, even over the long term, new research shows."In FRIDAY, Nov. In the present study, approximately 10 years after 48 months of.
Treatment typically involves surgery and follow-up hormone therapy, but late effects of these treatments include osteoporosis, sexual dysfunction and blood clots. Despite significant therapeutic advances, breast cancer remains a leading cause of cancer-related death in women.
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