A personalized gene editing therapy was successfully used to treat an infant with CPS1 deficiency, a disease that causes ammonia buildup in the blood. The technology used creates an opportunity for the development of personalized treatments for other rare diseases.
Healthcare providers expressed concerns about the accuracy gaps in AI image analysis for non-white skin tones or low digital literacy, creating potential health disparities.
Healthcare providers are exploring ways to reduce patient travel through a growing trend of expanding sites of administration beyond academic medical centers into community and outpatient settings closer to patients' homes.
Patients who were positive for cytomegalovirus had more immune cells circulating in their blood before they started immunotherapy. This may be one reason why these patients responded better to certain immunotherapy treatments, researchers said.
Plans expect cell and gene therapies to pose a moderate or major financial challenge in the next two to three years, according to a new survey of pharmacy benefit leaders by PSG.
In mice with human Merkel cell carcinoma, pyrvinium pamoate significantly reduced tumor growth, potentially offering a new way to treat this aggressive skin cancer.
A phase 1b showed promising results in lowering LDL in patients with heterozygous familial hypercholesterolemia and/or premature coronary artery disease.
A new study suggests that targeting the protein alpha-synuclein could be a possible new target for treating melanoma.
Lexeo Therapeutics’ gene therapy provides a healthy version of the frataxin gene, which is critical for energy production in cells. The company plans to begin a pivotal trial next year.
Patients with a rare disease called mucous membrane pemphigoid are at risk of developing squamous cell carcinoma and basal cell carcinoma, finds a new study.
None of the cell and gene therapies that reached the final regulatory submission stage were rejected by the FDA.
The Map My Mole app provides patients with a small lens attachment for their phones to capture high-quality images of concerning skin spots. These images are then uploaded for assessment by consultants.
Encelto is small, semi-permeable capsule implanted in the eye that contains allogeneic retinal pigment epithelium cells genetically engineered to produce specific therapeutic proteins. It will be available in June 2025.
EsoBiotec’s platform has the potential to eliminate the complex and time-consuming process for manufacturing, helping to make CAR-T cell therapies more accessible.
Early research shows a one-time gene therapy has potential for addressing both the liver and lung disease that patients with alpha-1 antitrypsin deficiency (AATD) face.
Researchers said alpha particles are promising therapies that target cancer while minimizing the damage to nearby healthy tissues.
Deborah S. Sarnoff, M.D., talks about the recent breakthrough treatments that are paving the way to treat patients with advanced basal cell carcinoma, squamous cell carcinoma and Merkel cell carcinoma, which is a rare and aggressive type of skin cancer.
In a small study in a community oncology practice, more than 60% of patients with advanced squamous cell skin cancer treated with Libtayo achieved complete remission.
Researchers have suggested that uniform evidence standards and reporting requirements could help speed patient access to cell and gene therapies and help ensure consistent safety and efficacy evaluations.
Solid Biosciences’ gene therapy uses a shortened version of the dystrophin protein, which is engineered to mimic the key functions of full-length dystrophin and allows it be delivered more effectively to muscle cells.
Risk factors for secondary skin cancer after blood or marrow transplant include those who had treatment with a monoclonal antibody, chronic graft-versus-host disease and posttransplant immunosuppression.
A mouse study at Memorial Sloan Kettering Cancer Center has found that adding Nef, a protein from HIV, allows donor CAR T cells to avoid detection by the immune system while still attacking tumors.
A novel AI model was able to achieve 99.86% accuracy for diagnosing skin conditions, even in people with darker skin tones or excessive hair.
Becky Kamowitz was appointed executive director of The Skin Cancer Foundation in January 2025. She talks about the organization’s priorities for the coming year.
President Trump signed an executive order on the day of his inauguration that created doubts about the future of a program that helps state Medicaid programs pay for expensive cell and gene therapy.
In a review of previous studies, researchers found that the images seen on social media contribute to taking actions that are both beneficial with patients seeking dermatological care and unfavorable with increased exposure to UV radiation for a better tan.
A small study of 18 patients with skin cancer found that the combination of vitamin D and chemotherapy provided an immune response that lasted up to five years.
Few studies in this analysis assessed the accuracy of AI in detecting melanoma in diverse skin tones.
Some muscles are resistant to the loss of motor neurons seen in patients with spinal muscular atrophy, and new research has discovered that even in muscles that appeared resistant to SMA, subtle changes had occurred at the cellular level.
