First- or best-in-class programs with compelling preclinical efficacy already demonstrated

Our programs are underpinned by world-class translational science, a deep understanding of disease biology, and real-world clinical appreciation of each disease indication stemming from the close collaboration between our academic founders. By targeting the root cause of each disease, we aim to bring potentially curative benefits to patients.

Target	Program	Discovery	Preclinical	Clinical	Caption
Liver	BGT-OTCD	100	100	5	Ornithine Transcarbamylase Deficiency (OTCD)
CNS	BGT-DTDS	100	90		Dopamine Transporter Deficiency Syndrome (DTDS)
CNS	BGT-NPC	100	80		Niemann-Pick Disease Type C (NPC)
CNS	BGT-INAD	100	80		Infantile Neuroaxonal Dystrophy (INAD)

Target

Program

Discovery

Preclinical

Clinical

Caption

Liver

BGT-OTCD

100

Ornithine Transcarbamylase Deficiency (OTCD)

CNS

BGT-DTDS

100

Dopamine Transporter Deficiency Syndrome (DTDS)

CNS

BGT-NPC

100

Niemann-Pick Disease Type C (NPC)

CNS

BGT-INAD

100

Infantile Neuroaxonal Dystrophy (INAD)

First- or best-in-class programs with compelling preclinical efficacy already demonstrated

Pipeline overview