CHOP at ASGCT, Treatment for Friedreich’s Ataxia, New Nephrology Chief

In our latest roundup of Children's Hospital of Philadelphia research news, read highlights from the 25th Annual American Society of Gene and Cell Therapy (ASGCT) Meeting, learn about a newly designated drug for treatment of Friedreich's Ataxia, and meet CHOP's new Chief of the Division of Nephrology.

CHOP Researchers Present at 25th Annual ASGCT Meeting

Children's Hospital of Philadelphia researchers gathered virtually and in person May 16-19 in Washington D.C. for the 25th annual ASGCT Meeting. As the primary membership organization for cell and gene therapy science, ASGCT brings together scientists, physicians, patient advocates, and other stakeholders to advance the discovery and clinical application of these novel treatments to alleviate human disease. This year's meeting highlights include a number of research abstracts presented by CHOP scientists, new awards, and elections of CHOP principal investigators, demonstrating the Research Institute's leading role in the cell and gene therapy space.

• An active member since ASGCT's founding, Beverly Davidson, PhD, Chief Scientific Strategy Officer, completed her term as ASGCT president for 2021-2022.
• Lindsey George, MD, investigator in the Division of Hematology, was elected to the ASGCT Board of Directors, and received the Outstanding New Investigator Award.
• Katherine High, MD, CHOP Gold Medal recipient and founder of the , received the Jerry Mendell Award for Translational Science.

A number of CHOP investigators also presented their research through oral abstract sessions and lectures:

• William Peranteau, MD, presented on in utero gene editing for congenital disease.
• Denise Sabatino, PhD, presented on understanding and mitigating the potential for AAV-associated oncogenesis.
• Lindsey George presented on the enhanced function of intrinsic tenase proteins for hemophilia gene therapy.
• Alejandro Mas Monteys, PhD, presented on the Xon system, a drug-induced splicing switch for regulated control of gene therapies.
• Joseph Rossano, MD; Can Ficicioglu, MD, PhD; Stefano Rivella, PhD; Dr. Davidson; Elizabeth Bhoj, MD, PhD; Adeline Vanderver, MD; Rebecca Ahrens-Nicklas, MD, PhD; and Leyuan Ma, PhD, presented oral and poster abstracts related to cell and gene therapy in their respective research specialties.

Visit the ASGTC for the complete 2022 abstract collection.

David Lynch, MD, PhD

FDA Grants Special Designation to Friedreich's Ataxia Treatment

A potential new therapy for the progressive neurological disorder, Friedreich's Ataxia (FA) was granted Fast Track Designation and Orphan Drug Designation by the U.S. Food and Drug Administration based on study findings led by a CHOP neurologist.

The designation puts the treatment, called omaveloxolone, on an expedited process to become the first therapy approved for patients with FA. An oral, once-daily drug, omaveloxolone works by activating Nrf2, a transcription factor that induces molecular pathways that promote the resolution of neuroinflammation by restoring mitochondrial function, reducing oxidative stress, and inhibiting pro-inflammatory signaling.

In collaboration with the biopharmaceutical company Reata Pharmaceutical and other colleagues around the globe, David Lynch, MD, PhD, director of the FA Program at CHOP, published results that show omaveloxolone has a potentially beneficial effect in slowing the progression of FA out to more than two years. In their study published in Neurodegenerative Disease Management, the researchers show that omaveloxolone modifies the long-term behavior of FA, and when analyzed in a delayed-start fashion, each trial cohort benefitted equally from the drug no matter when they started omaveloxolone.

Learn more.

Benjamin, Laskin, MD, MS

CHOP Appoints New Chief of Nephrology

Join us in congratulating Benjamin, Laskin, MD, CHOP’s new chief of the Division of Nephrology in the Department of Pediatrics! An investigator and physician at CHOP for the past 11 years, Dr. Laskin's research is focused on infections and kidney disease in immunosuppressed children, particularly those receiving a bone marrow or organ transplant. His work on preventing, treating, and slowing the progression of kidney disease in children receiving a hematopoietic cell transplant (HCT) is extensive. As principal investigator on a National Institutes of Diabetes and Digestive and Kidney Diseases research study, he leads a project testing the association between viral infection and kidney and bladder disease in children receiving an allogenic HCT. In addition, he has published numerous studies on infections after kidney transplant and HCT, and the study of thrombotic microangiopathy after HCT.

As Chief of Nephrology, Dr. Laskin will continue to work with a multidisciplinary team at CHOP to provide and advance high-quality care for children with kidney disease or hypertension.

"I am delighted to announce the well-earned appointment of Dr. Benjamin Laskin to Chief of our Division of Nephrology," stated Joseph St. Geme, MD, Physician-in-Chief at CHOP in a press release. "Dr. Laskin is a highly accomplished physician, educator, and scholar with an impressive background in nephrology. I look forward to watching the division grow to an even higher level of excellence under Dr. Laskin's leadership. Additionally, we thank Dr. Madhura Pradhan for her time as interim Chief of Nephrology and are eager to see her continue her important roles in the division and across CHOP."

Learn more.

ICYMI

Catch up on our headlines from our June 24 In the News:

• CRISPR Gene Editing Therapy Shows Continued Promise in Blood Disorders
• High-Dose Celecoxib Reduces Narcotic Use, Controls Pain After Tonsillectomy
• JCEMHighlights Research Led by CHOP Researchers
• Researchers Reveal Molecular Mechanics of Potential Immunotherapy Target
• First CAR T-Cell Therapy Demonstrates Benefits Five Years Later

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