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Programs

Putting our science into action

We are working to bring our approach and the possibility of a cure to patients with high-risk Acute Myeloid Leukemia (AML) and other Blood Cancers.

Our research programs

Trem-cel + Mylotarg

Trem-cel (tremtelectogene empogeditemcel) is a shielded stem transplant in development for patients with AML and MDS, in which healthy transplant donor cells are genetically engineered by removing CD33. Patients are then treated post-transplant with Mylotarg™ to selectively kill cancer cells and avoid toxicity.

VCAR33ALLO

We are developing a highly potent CAR-T, the first healthy donor HLA-matched CD33-directed CAR-T in the clinic for AML, which could be potentially curative.

Trem-cel + VCAR33 Treatment System

This Treatment System combines trem-cel and VCAR33ALLO representing another significant synergistic treatment option that could potentially cure AML.

VADC45

VADC45 is a CD45-directed ADC. CD45 is an excellent target for a variety of blood cancers with clinical proof-of-concept.

CD33-CLL1 Multiplex Treatment System

This Treatment System would combine a shielded transplant removing multiple surface targets with a dual-specific CAR-T that combines two constructs that target the two most promising antigens in AML aiming for deeper and more durable responses in relapsed/refractory AML.

Discovery platform

We are conducting ongoing discovery efforts in commonly transplanted hematologic malignancies.

Manufacturing

We operate an in-house clinical manufacturing facility in Cambridge, Massachusetts to support clinical development of our therapeutic candidates for patients with blood cancers.