Cell therapy weekly: US FDA grants Orphan Drug Designation to a gene therapy for a type of retinal disease
This week: The US Food and Drug Administration (FDA; MD, USA) grants Fast Track designation for an autologous Treg cell therapy for multiple sclerosis (MS) and granted Orphan Drug Designation to a gene therapy for retinitis pigmentosa. Plus, encouraging results from a B-cell CAR-T therapy trial for CD19+ acute lymphoblastic leukemia.
The news highlights:
- Treg therapy receives FDA Fast Track designation
- Encouraging results from B-cell acute lymphoblastic leukemia CAR-T therapy trial
- Gene therapy for retinal disease receives FDA Orphan Drug Designation
Treg therapy receives FDA Fast Track designation
The FDA has granted Fast Track designation to Abata Therapeutics’ (MA, USA) autologous Treg therapy for progressive MS. The therapy, ABA-101, also recently received Investigational New Drug application clearance from the FDA.
ABA-101 utilizes a patient’s Treg cells that have been engineered to express a T-cell receptor that targets immunogenic myelin fragments in the central nervous system, reducing inflammation at the site of the disease.
“There are no effective treatments for progressive MS and rapidly advancing new therapies is critical for patients and their families. We are very pleased that the FDA granted us Fast Track designation as it will enable us to expedite our efforts to bring ABA-101 to patients,” said Samantha Singer, CEO of Abata Therapeutics. “We are focused on initiating our Phase I study this year in patients and evaluating the potential impact of this important new therapy.”
Encouraging results from B-cell acute lymphoblastic leukemia CAR-T therapy trial
Orgenesis Inc. (MD, USA) has detailed encouraging data from its real-world study of ORG-101, a CD19 CAR-T therapy, in patients with CD19+ acute lymphoblastic leukemia, also termed B-cell ALL.
Among the study’s findings, 82% of adult patients and 93% of pediatric patients exhibited complete response to the therapy. In addition, the study illustrated low incidence of severe cytokine release syndrome, a common safety concern with CAR-T therapies, in both adult and pediatric patients.
The company plans to commence multicenter Phase I/II trials through the support of its Enterprise Greece Grant whilst leveraging its recently acquired good manufacturing practice-validated platform, which has been developed to efficiently fast-track production of CAR-T therapies and reduce the therapy’s costs.
“We believe that these clinical results combined with our [good manufacturing practice-validated platform] are a significant step forward for our strategy to combine our strong capabilities in decentralized cell therapy production with our regional partnerships,” said Vered Capla, CEO at Orgenesis. “Not only has the product shown initial signs of positive clinical outcomes, but our production data also validated that Orgenesis’ cost-effective decentralized cell processing has the potential to improve access to this treatment and reduce costs. We remain committed to bringing this and other potentially life-saving treatments to patients in need worldwide.”
Gene therapy for retinal disease receives FDA Orphan Drug Designation
SKG1108, a one-time gene therapy for the treatment of retinitis pigmentosa, has been granted Orphan Drug Designation from the FDA. SKG1108, developed by Skyline Therapeutics (Shanghai, China), is manufactured utilizing a recombinant adeno-associated virus to directly deliver single-stranded DNA encoding light-activatable proteins to the retina, to generate new photo-sensing cells, resulting in improved visual function.
Retinitis pigmentosa is a genetic retinal disease that gradually but progressively leads to vision impairment, often resulting in blindness by age 40. Typically, the disease starts with the decline of rod cells, followed by the loss of cone cells. While treatment options are currently limited, the development of SKG1108 offers the potential for vision restoration in a wider range of patients, regardless of the genetic abnormalities that cause the disease.
