Datar Cancer Genetics Unveils Exacta AI: A Revolutionary AI-Driven Cancer Treatment Platform

Metropolis Healthcare Partners with Roche to Launch Self-Sampling HPV DNA Test for Cervical Cancer Screening in India

Union Budget 2025: Full Exemption from Basic Customs Duty (BCD) for 36 Lifesaving Drugs

Dr. Jitendra Singh Inaugurates Centre for Advanced Genomics & Precision Medicine at AIIMS Jammu

Union Budget 2025-26: Key Healthcare Measures for Cancer Care and Access to Life-Saving Drugs

FDA Approves Roche’s PATHWAY HER2 Test for Identifying HER2-Ultralow Breast Cancer Patients Eligible for ENHERTU

Bristol Myers Squibb’s Breyanzi® Receives CHMP Recommendation for Relapsed or Refractory Follicular Lymphoma in the EU

NHS to Offer Groundbreaking Gene Therapy for Sickle Cell Disease

Quibim Secures $50M Series A to Advance AI-Powered Imaging for Precision Medicine

Apollo Hospitals Unveils Advanced Oncology Facility in Chennai

Beckman Coulter’s Alzheimer’s Blood Test Receives FDA Breakthrough Device Designation

ANGLE Announces Breakthrough Dual CTC-DNA and ctDNA Analysis with Illumina’s NGS Solution

Diatech Pharmacogenetics and Merck Expand Collaboration to Enhance RAS Biomarker Testing in MEA Region

DCG Unveils AI-Powered Video-Reporting for Personalized Chemotherapy Selection

FDA Approves Monthly Maintenance Dosing for LEQEMBI® in Alzheimer’s Treatment

Home
/
update
/
details

FDA Breaks New Ground with Approval of Two Gene Therapies for Sickle Cell Disease, Including Revolutionary CRISPR Drug

NextEdge Admin

10 Nov 2023

2 min 51 sec

The US Food and Drug Administration (FDA) has granted approval for two groundbreaking gene therapies aimed at treating sickle cell disease (SCD). This decision marks a significant milestone, with one of the therapies, Casgevy (exagamglogene autotemcel), developed by Vertex Pharmaceuticals and CRISPR Therapeutics, being the first-ever drug to gain approval utilizing CRISPR gene-editing technology. Casgevy involves editing patients’ own CD34-positive hematopoietic stem cells using CRISPR-Cas9 technology outside the body to modify the BCL11A gene. This alteration increases the production of fetal hemoglobin, addressing the dysfunction observed in SCD patients. The other approved therapy, Lyfgenia (lovotibeglogene autotemcel), developed by Bluebird Bio, entails genetically modifying patients’ own hematopoietic stem cells using lentiviral vectors. This modification delivers a functional copy of a modified beta-globin gene, enabling the production of anti-sickling hemoglobin (HbAT87Q), functioning similarly to normal adult hemoglobin.

Both therapies are one-time infusions made from the patient’s own blood stem cells and are authorized for individuals aged 12 years and older with a history of vaso-occlusive crises. The FDA highlighted the rigorous evaluation of scientific and clinical data supporting the approval, emphasizing its commitment to advancing safe and effective treatments for severe health conditions. In addition to the approval, the FDA included a black box warning for Lyfgenia, emphasizing the need for life-long monitoring due to hematologic malignancies observed in some treated patients. Both Casgevy and Lyfgenia will undergo long-term studies to assess their safety and effectiveness. Common side effects reported for Casgevy included low platelet and white blood cell levels, mouth sores, nausea, and musculoskeletal pain, while Lyfgenia exhibited side effects such as stomatitis, febrile neutropenia, and low blood cell counts. Both therapies had previously received orphan drug, fasttrack, and regenerative medicine advanced therapy designations from the FDA