FDA Approves Groundbreaking Cell-Based Gene Therapies for Sickle Cell Disease

FDA Approves Groundbreaking Cell-Based Gene Therapies for Sickle Cell Disease

Written by Stacy M. Brown

 

Sickle cell disease, a group of inherited blood disorders, affects around 100,000 individuals in the United States and is predominant among African Americans.

The U.S. Food and Drug Administration (FDA) has greenlit two revolutionary cell-based gene therapies, Casgevy and Lyfgenia, marking a significant leap forward in treating sickle cell disease (SCD) for patients aged 12 and older.
The approval by the FDA signifies the commencement of a novel epoch in managing sickle cell disease, providing optimism to individuals whose lives have been significantly disrupted by the arduous condition.

Sickle cell disease, a group of inherited blood disorders, affects around 100,000 individuals in the United States and is predominant among African Americans. Health officials said the root cause of SCD is a mutation affecting hemoglobin, a crucial protein in red blood cells responsible for oxygen delivery. The genetic problem causes red blood cells to have a unique “sickle” shape, which can lead to vaso-occlusive events (VOEs) or vaso-occlusive crises (VOCs), which are very painful and damage organs. The recurrence of these crises poses life-threatening risks and potential disabilities.

“Sickle cell disease is a rare, debilitating, and life-threatening blood disorder with significant unmet need, and we are excited to advance the field,” said Nicole Verdun, M.D., director of the Office of Therapeutic Products within the FDA’s Center for Biologics Evaluation and Research.
Casgevy, a groundbreaking cell-based gene therapy, is the first FDA-approved treatment employing CRISPR/Cas9, a revolutionary genome editing technology. The therapy is for individuals 12 years of age or older who have recurrent vaso-occlusive crises. It changes the patient’s hematopoietic stem cells using CRISPR/Cas9, a technology that can precisely edit DNA.

The edited cells are then transplanted back into the patient, enhancing the production of fetal hemoglobin and preventing the sickling of red blood cells.
Lyfgenia is another cell-based gene therapy that uses a lentiviral vector to change genes. The FDA approved it for those 12 years of age or older who have SCD and a history of vaso-occlusive events. Lyfgenia changes blood stem cells to make HbAT87Q, gene-therapy-derived hemoglobin that looks like adult hemoglobin and makes it less likely that red blood cells will sickle. Both therapies utilize the patients’ blood stem cells, administered through a one-time, single-dose infusion following myeloablative conditioning.

“These approvals represent an important medical advance with the use of innovative cell-based gene therapies to target potentially devastating diseases and improve public health,” said Dr. Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research.
The Casgevy and Lyfgenia applications received Priority Review, Orphan Drug, Fast Track, and Regenerative Medicine Advanced Therapy designations. Casgevy was granted approval to Vertex Pharmaceuticals, Inc., and Lyfgenia to Bluebird Bio, Inc.

The FDA said its approval of Casgevy was based on a single-arm, multicenter trial evaluating its safety and effectiveness in adult and adolescent SCD patients. Of the 44 treated patients, 93.5% achieved freedom from severe VOC episodes for at least 12 consecutive months. Common side effects included low platelet and white blood cell levels, mouth sores, nausea, and musculoskeletal pain.

Lyfgenia’s approval was based on a 24-month multicenter study, with 88% of patients achieving complete resolution of VOEs between 6- and 18-months post-infusion. Side effects included stomatitis, low blood cell levels, and febrile neutropenia. A black box warning highlighting the risk of hematologic malignancy accompanies Lyfgenia’s label, emphasizing the need for lifelong monitoring in patients.

“Today’s actions follow rigorous evaluations of the scientific and clinical data needed to support approval, reflecting the FDA’s commitment to facilitating the development of safe and effective treatments for conditions with severe impacts on human health,” Dr. Marks asserted.

October 16, 2023, HOUSTON, TX – Congressional Candidate Amanda Edwards has raised over $1 million in less than 4 months, a substantial sum that helps bolster the frontrunner status of the former At-Large Houston City Council Member in her bid for U.S. Congress. Edwards raised over $433,000 in Q3 of 2023. This strong Q3 report expands on a successful Q2 where Edwards announced just 11 days after declaring her candidacy that she had raised over $600,000. With over $829,000 in cash-on-hand at the end of the September 30th financial reporting period, Edwards proves again that she is the clear frontrunner in the race. “I am beyond grateful for the strong outpouring of support that will help me to win this race and serve the incredible people of the 18th Congressional District,” said Edwards. “We are at a critical juncture in our nation’s trajectory, and we need to send servant leaders to Congress who can deliver the results the community deserves. The strong support from our supporters will help us to cultivate an 18th Congressional District where everyone in it can thrive.” Edwards said. “Amanda understands the challenges that the hard-working folks of the 18th Congressional District face because she has never lost sight of who she is or where she comes from; she was born and raised right here in the 18th Congressional District of Houston,” said Kathryn McNiel, spokesperson for Edwards’ campaign. Edwards has been endorsed by Higher Heights PAC, Collective PAC, Krimson PAC, and the Brady PAC. She has also been supported by Beto O’Rourke, among many others. About Amanda: Amanda is a native Houstonian, attorney and former At-Large Houston City Council Member. Amanda is a graduate of Eisenhower High School in Aldine ISD. Edwards earned a B.A. from Emory University and a J.D. from Harvard Law School. Edwards practiced law at Vinson & Elkins LLP and Bracewell LLP before entering public service. Edwards is a life-long member of St. Monica Catholic Church in Acres Homes. For more information, please visit www.edwardsforhouston.com

As September 13th rolls around, we extend our warmest birthday wishes to the creative powerhouse, Tyler Perry, a man whose indomitable spirit and groundbreaking work have left an indelible mark on the world of entertainment. With his multifaceted talents as an actor, playwright, screenwriter, producer, and director, Tyler Perry has not only entertained but also inspired audiences worldwide, particularly within the African-American community, where his influence and role have been nothing short of powerful. Born in New Orleans, Louisiana, in 1969, Tyler Perry’s journey to stardom was a path riddled with adversity. Raised in a turbulent household, he found refuge in writing, using it as a therapeutic outlet. This period of introspection gave rise to one of his most iconic creations, Madea, a vivacious, no-nonsense grandmother who would later become a beloved figure in Perry’s works, offering a unique blend of humor and profound life lessons. Despite facing numerous challenges, including rejection and financial struggles, Perry’s determination and unwavering belief in his abilities propelled him forward. In 1992, he staged his first play, “I Know I’ve Been Changed,” which, although met with limited success, was a pivotal moment in his career. Unfazed by initial setbacks, Perry continued to hone his craft, and by 1998, he had successfully produced a string of stage plays that showcased his storytelling prowess.

Calling all teenage student-athletes! If you have dreams of playing college soccer and wish to represent an HBCU, the HBCU ID Camp is your golden opportunity. From 8 am to 5 pm on November 11-12, Houston Sports Park will transform into a hub for aspiring male and female soccer players. Coaches from HBCUs across the nation will be present to evaluate, scout, and offer valuable feedback. Moreover, they might even spot the next soccer prodigy to join their collegiate soccer programs. This camp is not just about honing your soccer skills but also a chance to connect with the HBCU soccer community. You’ll learn the ins and outs of what it takes to excel on the field and in the classroom, which is crucial for a college athlete. The HBCU ID Camp is an excellent platform to network with coaches, learn from experienced athletes, and take the first steps toward your college soccer journey. To secure your spot at this incredible event, don’t forget to register [here](insert registration link). Space is limited to 120 participants, so make sure to reserve your place before it’s too late. It’s time to turn your dreams of playing college soccer into a reality.

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