UNIVERSITY OF ALABAMA AT BIRMINGHAM

Towana Looney received a gene-edited pig kidney transplant at NYU Langone Health, marking a breakthrough in xenotransplantation. The procedure, involving 10 gene edits, aims to address the organ supply crisis. Looney, who had been on dialysis for years, is now in better health and the first to receive such a kidney.

An Alabama woman, Towana Looney, received a gene-edited pig kidney transplant at NYU Langone Health, marking a significant breakthrough in xenotransplantation. Looney, who had been on dialysis for years due to kidney failure, is now the healthiest she's been in eight years. The procedure involved 10 gene edits to the pig kidney, aiming to reduce rejection and improve compatibility. This is the third time a gene-edited pig kidney has been transplanted into a living human, and Looney is the first to receive a kidney with 10 gene edits.

United Therapeutics announced the first UKidney transplant into a living person on Nov 25, 2024, following successful UHeart and UThymoKidney transplants. The patient, Towana Looney, had kidney failure and couldn't find a suitable donor. The UKidney, a pig kidney with 10 gene edits, aims to address organ shortages.

Mohanraj Thirumalai, Ph.D., uses Azure AI to detect bias in research studies, ensuring inclusivity for people with disabilities. His work involves analyzing over half a million records to identify and address exclusionary practices, presenting findings on an interactive dashboard to inform stakeholders. Thirumalai envisions AI influencing policy changes and enhancing research inclusivity.

Nita Limdi, PharmD, Ph.D., MSPH, FAHA, has been named associate dean for Genomic Medicine at UAB, succeeding Bruce Korf. She will lead efforts to grow and expand UAB's Genomic Medicine program through interdisciplinary collaborations, securing external grant funding, and enhancing national and international partnerships. Limdi's research focuses on pharmacogenomics and genomics of anticoagulation therapy, earning her the 2022 Heersink School of Medicine Dean’s Excellence Award for Research. She has extensive experience in mentoring and has contributed to greater diversity in clinical research and implementation.

Arlocabtagene autoleucel (arlo-cel) showed a 48% CR rate in heavily pretreated relapsed/refractory multiple myeloma patients at the RP2D, with an ORR of 91% in those receiving 150 x 10^6 cells. Responses were high across high-risk subgroups, including triple class refractory disease, and were not affected by prior BCMA-targeted therapy. The median duration of response was 18.0 months, and 38% of responses were ongoing at 16.1 months follow-up. The therapy demonstrated manageable safety and has received FDA's regenerative medicine advanced therapy designation.

Sanofi's rilzabrutinib shows promise in treating immune thrombocytopenia, while Novo Nordisk's etavopivat reduces sickle cell pain crises. Beam Therapeutics' CRISPR therapy for sickle cell yields consistent results, though fertility preservation remains a concern.

Novo Nordisk announced 52-week results from the phase 2 HIBISCUS study of etavopivat in sickle cell disease, showing a trend toward reduced vaso-occlusive crises and increased hemoglobin response. Results will be confirmed in the phase 3 HIBISCUS trial.

Phase 2 data from the HIBISCUS study indicates etavopivat may reduce vaso-occlusive crises in sickle cell disease, with a trend towards lower VOC incidence and increased hemoglobin response. Results will be further tested in the phase 3 HIBISCUS trial.