
Phase 3 studies are continuing in the enhanced Padua FIX variant AMT-061 for hemophilia B.

Phase 3 studies are continuing in the enhanced Padua FIX variant AMT-061 for hemophilia B.

The executive vice president of research and development at Jasper Therapeutics discussed positive data from trials of JSP191 in MDS and AML.

The chief executive officer of CG Oncology discussed the role of CG0070 in patients who are unresponsive and responsive to Bacillus Calmette-Guerin immunotherapy.

The designations allow for expedited drug development and review of the investigational gene therapies for CLN2 Batten disease and SOD1 ALS.

The clinical associate professor from the Rogel Cancer Center at Michigan Medicine discussed the future of CAR T-cell therapy in mantle cell lymphoma patients.

Manuel Litchman, MD, the president, chief executive officer, and director of Mustang Bio, discussed upcoming research on MB-101 cell therapy.

The lymphoma service chief at Memorial Sloan Kettering Cancer Center discussed the need for new therapies to treat diffuse large B-cell lymphoma.

Loretta Nastoupil, MD, and John Burke, MD, discussed treating relapsed/refractory (R/R) diffuse large B-cell lymphoma (DLBCL).

Short-term morphological rescue was seen after voretigene neparvovec-rzyl treatment.

The executive vice president of research and development at Jasper Therapeutics discussed how JSP191 is designed to be better-tolerated than current conditioning regimens.

Abeona Therapeutics is seeking a path towards BLA filing based on the promising clinical data.

NT-501 previously received orphan drug and fast track status from the FDA and Neurotech will pursue approval in late 2022.

The company plans to use the mRNA platform it harnessed for its COVID-19 vaccine to develop the vaccine for malaria.

The first patient in CG Oncology's phase 2 study of CG0070 was dosed in December 2020.

Investigators found that reactivating the CaMKII enzyme could protect against further vision loss.

Bluebird Bio plans to submit its biologics license application in the US by mid 2021.

The professor of internal medicine in the Division of Oncology and Hematology at the University of Nebraska Medical Center discussed the benefits of CAR T-cell for patients with follicular lymphoma.

The chief technology and chief executive officer of Mustang Bio discussed the company’s integrated approach to drug development.

Experts discussed their recommendations for second-line treatment options for patients with DLBCL.

The co-founder, president, and chief executive officer of Solid Biosciences, whose own son has DMD, discussed challenges in developing gene therapies for DMD.

Stephen J. Schuster, MD, discussed tisagenlecleucel's efficacy and safety over other available treatments for relapsed/refractory FL.

The chief executive officer and co-founder of Flexion Therapeutics discussed the company’s pain-focused pipeline, which includes a gene therapy that targets inflammation.

A drop in endogenous Factor VIII expression was observed from treatment to 5-year follow-up despite continued demonstration of efficacy.

Joseph Sullivan, MD, and Elaine C. Wirrell, MD, discuss new developments for the treatment of Dravet syndrome beyond seizure control including gene editing and therapy.

The chief technology officer of Mustang Bio discussed the company’s approach and future plans in CAR T production.

Encouraging results from the phase 1 BrainChild-01 trial were recently published.

Heterogeneity in the cellular and molecular features of CAR T-cell products contributes to variation in efficacy and toxicity follow treatment with axicabtagene ciloleucel.

huCART19 is designed to yield longer remission rates for pediatric and young adult patients with relapsed or refractory B-cell acute lymphoblastic leukemia.

The hematologist/oncologist from the Harold C. Simmons Comprehensive Cancer Center discussed the updated results of the KarMMa trial in relapsed/refractory multiple myeloma.

MCO-010 is designed to deliver multi-characteristic opsin to retinal cells.