Our programs span discovery-stage to late-stage development and cover a range of high-value indications.
We aim to pursue programs we believe could be first-in-class / best-in-class and where there is prior learning in human genetics or precedented human activity for a pathway of interest. We place a premium on learnings from the clinic, whereby a drug has established the relevance of a biological pathway contributing to disease outcome. Our approach is to pursue the best assets in a capital efficient manner and rapidly progress our programs through development, evaluating the unique biological advantage of our product candidates.

Pipeline

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Overview
We are developing SerpinPC, a specific inhibitor of activated protein C (APC), for the treatment of hemophilia A and hemophilia B. SerpinPC has been observed to be well-tolerated in the clinical setting, associated with promising reductions in bleeding rates, and has PK suitable for infrequent subcutaneous dosing without the need for factor replacement. SerpinPC has human genetic target validation and established proof of concept Phase 2a clinical data.

Reason to Believe in Target
Human Genetics Support

Epidemiology
~20,000 persons with hemophilia in the United States
450,000 estimated global prevalence
Disease
Hemophilia A, B

Overview
We aim to develop novel therapeutics based on our unique platform technology, which is designed to selectively drive potent effector function activity, such as CD47 or CD3, while avoiding systemic toxicity. The lead compound is LB101,  a conditionally tetravalent PD-L1xCD47 LockBody® bispecific monoclonal antibody, which has two anti-CD47 domains blocked by two anti-PD-L1 domains, with proprietary human IgG-derived hinges linking the anti-CD47 and anti-PD-L1 domains. The anti-CD47 domain is blocked by the PD-L1 tumor targeting domain until the IgG-derived hinges are naturally degraded in the tumor microenvironment (TME), thus unlocking and activating the CD47 effector function activity in the tumor. We are also developing LB201, a conditionally bivalent PD-L1xCD3 LockBody® bispecific monoclonal antibody, which is designed to selectively drive potent CD3 effector function activity while avoiding systemic toxicity.

Reason to Believe in Target
Human Genetics Support

Epidemiology
Up to ~19 million new cases and ~10 million deaths globally, including
~1.9 million and ~600,000 within the United States
Disease
Solid Tumors

Overview
We are developing MGX292, a recombinant modified BMP9 replacement protein designed to overcome the deficiency in BMP9 signaling in PAH, directly targeting a central underlying disease mechanism (BMP9/BMPR2/ALK1 signaling pathway genetically altered in PAH).

Reason to Believe in Target
Human Genetics Support

Epidemiology
PAH prevalence is 11 to 26 per million individuals, affecting approximately 70,000 patients in North America, Europe and Japan
~1.9 million and ~600,000 within the United States
Disease
Pulmonary Arterial Hypertension (PAH)

Overview
We are developing oral and intranasal selective orexin receptor 2 (OX2R) agonists designed to leverage unique structural insights and to directly target the underlying pathophysiology of orexin neuron loss in NT1. Potential expansion into Narcolepsy Type 2 (NT2), rare hypersomnias, and additional disorders.

Reason to Believe in Target
Precedented Clinical Activity

Epidemiology
Estimated narcolepsy prevalence of over ~150,000 in the United States, of which approx. ~50% have NT1
~3 million prevalence of narcolepsy worldwide
Disease
Narcolepsy Type 1

Overview
We are developing CBS001, a high-affinity anti-LIGHT antibody, which preferentially binds the inflammatory membrane form of LIGHT.

Reason to Believe in Target
Precedented Non-Clinical Activity

Overview
We are developing CBS004, a humanized mAb specific to BDCA2, which is expressed exclusively on plasmacytoid dendritic cells (pDCs).

Reason to Believe in Target
Precedented Human Activity

Epidemiology
~200 cases per 1 million adults worldwide (SSc)

~70 cases per 100,000 persons (CLE/SLE)

Disease
Autoimmune Diseases