News

Approach can unravel causes in MYOC and TBK1 glaucoma.

A marketing authorization application has been submitted to the European Medicines Agency for the approval of the CAR T-cell therapy ciltacabtagene autoleucel in the treatment of patients with relapsed and/or refractory multiple myeloma.

Patients with the GBA mutation of Parkinson disease (PD) were shown to exhibit more severe cognitive decline than patients with idiopathic PD and those with both the GBA and LRRK2 G2019S mutations of PD.

Sequential therapy with CD19.28ζ-directed CAR T cells followed by allogeneic hematopoietic stem cell transplant induced durable disease control in a significant population of children and young adult patients with relapsed/refractory B-cell acute lymphoblastic leukemia.

Remestemcel-L reduced mortality through 60 days in a prespecified group of ventilator-dependent patients below the age of 65 with COVID-19 and moderate or severe acute respiratory distress syndrome.

Apic Bio plans to initiate a phase 1/2 clinical trial in late 2021 or early 2022 as a multi-center, 3-part study to evaluate APB-102 in patients with SOD1-ALS mutations.

A patient enrolled in the phase 2 INFINITY trial exploring ADVM-022 for diabetic macular edema experienced a suspected unexpected serious adverse reaction, which has led to an unmasking of the study.

The FDA has granted an orphan drug designation to the cell therapy ITIL-168 as a potential treatment for patients with stage IIB to IV melanoma.

The CEO of Lineage Cell Therapeutics discusses their unique approach to addressing degenerative vision loss in dry age-related macular degeneration.

Ajeet Gajra, MD, MBBS, FACP, talks about identifying and removing the barriers for offering CAR T-cell therapy at the community practice level.

The FDA has lifted a clinical hold placed on an IND for VY-HTT01 in Huntington disease, allowing Voyager Therapeutics to initiate a phase 1/2 study for the agent later this year.

A policy allowing enforcement discretion for cellular-derived therapies, including stem cell-based products, will end on May 31, 2021, allowing the FDA to take more action against unapproved regenerative medicines.

The FDA has lifted a clinical hold on the development of etranacogene dezaparvovec for patients with hemophilia B.

After a more than 35-year pursuit, the licensing of a gene therapy product for treating hemophilia may be available within 2 years.

The gene therapy SAR439483 demonstrated promising early signs of activity in patients with Leber congenital amaurosis caused by biallelic mutations in GUCY2D.

Nilanjan Ghosh MD, PhD, highlights progress made with CAR T-cell therapies in B-cell lymphoma and some ongoing trials generating interest in the field.

The FDA granted a Regenerative Medicine Advanced Therapy designation to the allogeneic chimeric antigen receptor T-cell therapy as a potential treatment for patients with relapsed/refractory multiple myeloma.

The FDA approval of brexucabtagene autoleucel established CAR T-cell therapy as a treatment option for patients with relapsed/refractory mantle cell lymphoma.

In an AAN plenary talk, Mark H. Tuszynski, MD, PhD, detailed the work he and colleagues have done to push stem cell therapy from the lab to the clinic to improve care for spinal cord injury.

The FDA has granted a fast track designation to LX1001 as a potential treatment for adult patients with APOE4-associated Alzheimer disease.

Vertex Pharmaceuticals will take the lead on worldwide development, manufacturing, and commercializing of CTX001, with payments and split economics to CRISPR Therapeutics.

Betibeglogene autotemcel is being withdrawn from the German market following a breakdown in reimbursement conversations, which has resulted in bluebird reducing and reshaping its European workforce.

Given that some patients may need to travel out of state to access CAR T sites of care, some may not have a clear understanding of their insurance benefits, including requirements for out-of-state or out-of-network treatment, as well as adequate assistance with the costs of medical-related travel.

Three cell and gene therapies were among the drugs included in a specialty drugs pipeline forecast at the Academy of Managed Care Pharmacy 2021 meeting.

In a phase 1 study of the innate cell engager AFM13 pre-complexed with NK cells, all 4 patients with CD30-positive, relapsed/refractory Hodgkin lymphoma treated with the therapy achieved at least a partial response.

Saad Z. Usmani, MD, FACP, discusses the rapidly changing cellular therapy paradigm in relapsed/refractory multiple myeloma.

The first patient has been enrolled and treated with RGX-121 in the third cohort of a phase 1/2 study for patients up to 5 years old with severe MPS II.

RP-L201 continued to show promising preliminary efficacy for patients with severe LAD-I after additional follow-up in a phase 1/2 study.

The FDA has granted an orphan drug designation to CYNK-001 as a potential treatment for adult patients with recurrent glioblastoma multiforme.

Studying zebrafish helps unravel the mysteries of photoreceptor regeneration.