FDA Grants Fast Track Status to Treg Therapy for Multiple Sclerosis and Orphan Drug Designation to Gene Therapy for Retinitis Pigmentosa
核心洞察
Abata Therapeutics (搜索)' ABA-101, an autologous Treg cell therapy, received FDA Fast Track designation for progressive multiple sclerosis (搜索), aiming to reduce inflammation in the central nervous system.
Orgenesis Inc. (搜索) reported encouraging data from a real-world study of ORG-101, a CD19 (搜索) CAR-T therapy, showing high complete response rates in both adult and pediatric patients with B-cell ALL.
Skyline Therapeutics (搜索)' SKG1108, a gene therapy for retinitis pigmentosa (搜索), received FDA Orphan Drug Designation, offering potential vision restoration by delivering light-activatable proteins to the retina.
The FDA has granted Fast Track designation to Abata Therapeutics (搜索)' autologous Treg therapy, ABA-101, for progressive multiple sclerosis (搜索) (MS) and Orphan Drug Designation to Skyline Therapeutics (搜索)' SKG1108, a gene therapy for retinitis pigmentosa (搜索). These designations aim to expedite the development and review of therapies addressing significant unmet medical needs.
Abata Therapeutics' ABA-101 Receives Fast Track Designation
Abata Therapeutics (搜索), based in Massachusetts, announced that its ABA-101, an autologous Treg cell therapy, has received Fast Track designation from the FDA for the treatment of progressive MS. This designation is intended to facilitate the development and accelerate the review of drugs to treat serious conditions and fill an unmet medical need. ABA-101 also recently received Investigational New Drug application clearance from the FDA, paving the way for clinical trials.
ABA-101 is engineered to express a T-cell receptor that targets immunogenic myelin fragments in the central nervous system. By targeting these fragments, the therapy aims to reduce inflammation at the site of the disease, potentially slowing or halting the progression of MS. There are currently limited effective treatments for progressive MS, highlighting the urgent need for new therapeutic options.
"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 (搜索). "We are focused on initiating our Phase I study this year in patients and evaluating the potential impact of this important new therapy."
Orgenesis Inc. Announces Encouraging Results from CAR-T Therapy Trial
Orgenesis Inc. (搜索) has released encouraging data from its real-world study of ORG-101, a CD19 (搜索) CAR-T therapy, in patients with CD19+ acute lymphoblastic leukemia (搜索) (ALL), also known as B-cell ALL. The study demonstrated promising efficacy and safety profiles.
The study found that 82% of adult patients and 93% of pediatric patients exhibited a complete response to the therapy. Furthermore, the incidence of severe cytokine release syndrome (CRS), a common safety concern with CAR-T therapies, was low in both adult and pediatric patients. These results suggest that ORG-101 may offer a significant benefit to patients with B-cell ALL.
The company plans to initiate multicenter Phase I/II trials, supported by its Enterprise Greece Grant, utilizing its good manufacturing practice (GMP)-validated platform. This platform is designed to efficiently fast-track the production of CAR-T therapies and reduce costs, potentially improving access to this life-saving treatment.
"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."
Skyline Therapeutics' SKG1108 Receives Orphan Drug Designation
Skyline Therapeutics (搜索)' SKG1108, a one-time gene therapy for the treatment of retinitis pigmentosa (搜索), has been granted Orphan Drug Designation by the FDA. Retinitis pigmentosa is a genetic retinal disease that causes progressive vision loss, often leading to blindness by age 40. The disease typically begins with the degeneration of rod cells, followed by the loss of cone cells.
SKG1108 utilizes a recombinant adeno-associated virus (AAV) to deliver single-stranded DNA encoding light-activatable proteins directly to the retina. This approach aims to generate new photo-sensing cells, potentially restoring visual function in patients with retinitis pigmentosa (搜索), regardless of the underlying genetic abnormality. Current treatment options for retinitis pigmentosa are limited, making SKG1108 a promising potential therapy.