Pfizer has dosed its first patient in a phase 1 clinical trial of an investigational gene therapy for the treatment of patients with Duchenne muscular dystrophy, a genetic disease that results in the absence of dystrophin, a protein that helps to keep muscle cells intact.
On Thursday, GlaxoSmithKline (GSK) announced that it will be transferring its portfolio of approved and investigational gene therapies to Orchard Therapeutics in exchange for a 19.9% stake in the company.
How are community practices coping with administering chimeric antigen receptor (CAR)-T treatments? At the 2018 Community Oncology Conference hosted by the Community Oncology Alliance, Houston Holmes, MD, MBA, FACP, Texas Oncology, shared his experience with administering CAR T-cells in a community cancer center–based setting.
Pfizer announced that it has begun a Phase 1b clinical trial for PF-06939926, its mini-dystrophin gene therapy candidate, intended for boys with Duchenne muscular dystrophy.
CEO of Krystal Biotech discusses first topical gene therapy for dystrophic epidermolysis bullosa.
Ronny L. Jackson, MD, would give up $1 million in retirement income to head the Department of Veterans Affairs and work in the Trump administration; mega healthcare deals endanger independent practices of primary care providers; Novartis is buying a gene therapy firm focused on spinal muscular atrophy.
Despite advancements in treatment strategies for mantle cell lymphoma, patients experience diminishing responses from sequential lines of therapy as their disease progresses, sharpening the focus on developing new agents and combinations.
Jae H. Park, MD, discusses the impact of agents such as blinatumomab, inotuzumab ozogamicin, and tisagenlecleucel on the treatment of acute lymphoblastic leukemia.
New research suggests that the composition of bacteria in the gastrointestinal tract may help predict which patients with cancer may benefit most from adoptive T-cell immunotherapy, including chimeric antigen receptor T-cell therapy.
Enrollment for 2018 in Affordable Care Act plans is only slightly below figures from 2017; a rare breed of antibiotic-resistant bacteria is spreading in hospitals; a new company will develop off-the-shelf CAR T-cell therapies that do not need to be personalized to the patient.
At the NCCN Annual Conference, Dr. Bijal Shah of Tampa’s Moffitt Cancer Center highlighted ongoing challenges in administration of CAR T-cell therapy.
The FDA has granted a priority review to moxetumomab pasudotox for the treatment of adult patients with hairy cell leukemia who have received at least 2 prior lines of therapy.
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On the closing day of the National Comprehensive Cancer Network (NCCN) 23rd Annual Conference in Orlando, Florida, Bijal Shah, MD, of Moffitt Cancer Center, presented on acute lymphoblastic leukemia (ALL), and on the lessons learned from the application of chimeric antigen receptor (CAR) T-cell therapy in this indication.
Despite price tags well over $350,000 for treatment, tisagenlecleucel, approved for children with B-cell acute lymphoblastic leukemia, and axicabtagene ciloleucel, approved for adults with certain B-cell subtypes of non-Hodgkin lymphoma, are considered cost effective, according to a report from the Institute for Clinical and Economic Review.
The current standard-of-care therapy for patients with clear cell renal cell carcinoma is sunitinib, but many patients relapse or don't have responses to the drug.
Renier J. Brentjens, MD, PhD, discusses the emergence of BCMA as a target for CAR T-cell therapy and other next steps for the field.
A 40-year old patient at the UNC Clinical and Translational Research Center was treated with SB-913 this week.
This is the first and only FDA-approved gene therapy for treatment for an inherited disease.
The addition of weekly carboplatin following surgery and radiation therapy did not improve freedom from locoregional relapse in patients with high-risk cutaneous head and neck squamous cell carcinoma.
Both prior chemotherapy and a dependence on glycolysis appear to reduce the potential to develop T cells into chimeric antigen receptor T-cell therapy.
The market price for gene therapy greatly exceed the costs of the gene editing development and the equipment required for the task.
Abeona announced that the U.S. FDA has granted rare pediatric disease designation to ABO-202, a gene therapy in development for the treatment of infantile and late infantile-onset Batten disease.
Using CRISPR/Cas9 gene editing to remove CD7 from healthy T cells, researchers have found a way to use third party T cells for CAR-T therapy in T-cell hematologic malignancies.
Migraine is not characterized by neuronal degeneration, but it has been a target of stem cell therapy in a few small investigational studies--and has shown early promise.
Holger L. Gieschen, MD, discusses the latest developments with radiation therapy in non-small cell lung cancer.
Upfront testing for BRAF V600E mutations is necessary for patients with non–small cell lung cancer, but if results are positive, physicians are unsure when to administer BRAF/MEK combination therapy—and they must be prepared to manage the associated adverse events.
With durvalumab (Imfinzi) being hailed as a potentially practice changing therapy in locally advanced non–small cell lung cancer, ongoing studies are seeking to determine the efficacy of immunotherapy in earlier stages of disease.
Breakthrough gene therapy for B-cell lymphoma now available at Hackensack University Medical Center.
The fall of 2017 brought the approval of new gene therapies, and could usher in a new area of personalized medicine.
