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.

Target	Program	Discovery	Preclinical	IND-Enabling	Clinical	Caption
Liver	BGT-OTCD	100	100	100	20	Ornithine Transcarbamylase Deficiency (OTCD)
CNS	BGT-DTDS	100	100	90		Dopamine Transporter Deficiency Syndrome (DTDS)
CNS	BGT-PD	100	15			Parkinson’s Disease (PD)
CNS	BGT-NPC	100	90			Niemann-Pick Disease Type C (NPC)
CNS	BGT-INAD	100	90			Infantile Neuroaxonal Dystrophy (INAD)

Target

Program

Discovery

Preclinical

IND-Enabling

Clinical

Caption

Liver

BGT-OTCD

100

Ornithine Transcarbamylase Deficiency (OTCD)

CNS

BGT-DTDS

100

Dopamine Transporter Deficiency Syndrome (DTDS)

CNS

BGT-PD

100

Parkinson’s Disease (PD)

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