Latest Conference Coverage

The vice president of Public Policy & Advocacy of the Muscular Dystrophy Association discussed the organization’s work with regulatory members to advance gene therapies towards review and approval.

The vice president of Public Policy & Advocacy of the Muscular Dystrophy Association discussed the organization’s goals and strategies in educating regulatory decision-makers.

Schlechter discussed the potential advantages of TAC01-HER2 over current standards of care.

Schlechter discussed the design of the TACTIC-2 clinical trial for patients with HER2-positive solid tumors.

MRI revealed that participants who achieved sustained or confirmed disability improvement showed significantly less ventricular enlargement at 12 months.

As we learn more about genomics and identify more genes tied to rare disorders, the role of genetic counselors will become even more critical.

The senior vice president of Research & Early Development at Notch Therapeutics discussed the company’s approach to overcoming the drawbacks of autologous cell therapies.

Pam Gavin, executive vice president of NORD, discussed actions the organization is taking to further improve access to advanced care.

The executive director of Hemophilia Foundation Southern California discussed issues with minorities accessing care for rare diseases and strategies to mitigate them.

The professor of ophthalmology at the University College London Institute of Ophthalmology discussed the likely rise of gene therapies in inherited retinal diseases.

Participant 3 showed an 89% decrease in NAA in the CSF at 3 months post-treatment.

The chief scientific officer of cell therapy at Poseida Therapeutics discussed the company’s CAR-T and TCR technologies.

The chief medical officer of Triumvira Immunologics discussed the company’s future plans.

All 5 patients on ERT at study start have since been able to withdraw after gene therapy treatment.

Both botaretigene sparoparvovec and JNJ-81201887 were well-tolerated in treated patients, according to data from the 2022 AAO meeting.

The chief executive officer of Abintus Bio discussed the company’s in vivo approach and how it could help expand patient access to cell therapies.

The senior scientist at Thermo Fisher Scientific discussed the company’s approach to scaling and strategic partnerships.

Karen Walker, chief technology officer, Kyverna Therapeutics, discussed the company’s CAR T-cell and regulatory T-cell technologies.

Significant improvements were reported in full-field stimulus testing.

The chief executive officer of Cytoimmune discussed the company’s technology platforms and programs.

The vice president of oncology and research and development at Exuma Biotech discussed the company’s platforms, including CAR-TaNK cells.

Participants in the AAVIATE trial had up to an 85% decrease in treatment burden 6 months after treatment with RGX-314.

Rob Richards, administrative director, cell therapy and transplant, University of Pennsylvania, discussed improving reach of gene therapy trials and therapies.

The senior vice president of Technical Operations at Senti Biosciences discussed the company’s NK cell logic-gating platform and allogeneic approach.

Phil Cyr, Senior Vice President, Precision Value & Health, discussed challenges to tackle with CAR T-cell therapies.

The chief medical officer of Triumvira Immunologics discussed how TAC01-HER2 could address unmet needs in treating solid tumors.

The deputy director of the Masonic Cancer Center discussed trends of presentations from ESMO 2022.

Interim data showed a 78% overall response rate and a 67% complete response rate.