Vor Bio Doses First AML Patient with VCAR33 (ALLO) and Provides Corporate Update

• First patient dosed in company’s second clinical program with initial data expected in second half of 2024
  
• Cash runway extended into second half of 2025 with clinical trials on track

CAMBRIDGE, Mass., Jan. 17, 2024 (GLOBE NEWSWIRE) -- Vor Bio (Nasdaq: VOR), a clinical-stage cell and genome engineering company, announced today it has dosed the first patient in VBP301, its Phase 1/2, multicenter, open-label, first-in-human study of VCAR33^ALLO. The Company has extended its cash runway into the second half of 2025.

“We are pleased to start 2024 with strong execution and we look forward to sharing initial clinical data from the VBP301 trial later this year,” said Dr. Eyal Attar, Vor Bio’s Chief Medical Officer. “We are excited to bring to AML patients, for the first time, a healthy transplant donor-derived CAR-T developmental therapy which may overcome shortfalls seen with either autologous or allogeneic off-the-shelf approaches.”

VBP301 Clinical Trial/VCAR33^ALLO
The first patient with relapsed/refractory acute myeloid leukemia (AML) has been dosed with VCAR33^ALLO in the VBP301 clinical trial, a significant milestone demonstrating that VCAR33^ALLO can be successfully manufactured in Vor Bio’s in-house manufacturing facility.

VCAR33^ALLO is manufactured from lymphocytes collected from the patient’s original transplant donor, generating a CAR-T cell product that is exactly matched to the recipient’s engrafted blood system. By using healthy transplant donor cells as the starting material to produce VCAR33^ALLO, the CAR-T cells have a more stem-like phenotype, leading to greater potential for expansion, persistence, and anti-leukemia activity compared to a product derived from a patient’s own lymphocytes.

While this first patient had relapsed after a standard-of-care transplant, patients who have relapsed after a trem-cel transplant are also eligible to enroll in the VBP301 protocol and to receive VCAR33^ALLO. The ability to treat relapsed trem-cel transplant patients with VCAR33^ALLO may provide valuable early insights into the potential of the Company’s trem-cel + VCAR33 Treatment System, which pairs VCAR33 after trem-cel to reduce the risk of relapse or treat evidence of relapse. Initial data from VBP301 is expected in the second half of 2024.