Pipeline

New hope for patients with previously untreatable central nervous system disorders

A ROBUST PIPELINE FILLED WITH PROMISING GENE THERAPIES

Program*

Indication

Gene

Discovery
Pre-clinical
Phase 1/2
Pivotal
PEDIATRIC

Program*
PBGM01

Indication: GM1
gangliosidosis

Gene: GLB1

Details

Our gene therapy product candidate, PBGM01, utilizes an AAVhu68 viral vector to deliver a codon-optimized gene sequence encoding functional β-gal enzyme. The vector will be delivered directly to the cerebrospinal fluid by a single injection to the cisterna magna (ICM injection), with the goal of increasing β-gal enzyme activity levels in both the central nervous system and periphery.

Related link

Learn more about GM1 gangliosidosis

Program*
PBKR03

Indication: Krabbe disease

Gene: GALC

Details

Our gene therapy product candidate, PBKR03, utilizes an AAVhu68 viral vector to deliver a functional GALC enzyme gene that codes for galactosylceramidase (GAL-C). The vector will be delivered directly to the cerebrospinal fluid by a single injection to the cisterna magna (ICM injection), with the goal of increasing levels of the GALC enzyme in both the central nervous system (CNS) and the peripheral nervous system (PNS).

Related link

Learn more about Krabbe disease

Program*
PBML04

Indication: Metachromatic
leukodystrophy

Gene: ARSA

Details

We are developing PBML04 to treat metachromatic leukodystrophy (MLD), a recessive lysosomal storage disease caused by genetic mutations that result in demyelination of the fibers of the central and peripheral nervous systems, leading to progressive motor and cognitive impairments. We are developing PBML04 for patients who have MLD and mutations in the enzyme ARSA gene, which encodes the lysosomal enzyme ARSA.

Program*
PBCM06

Indication: Charcot-Marie-
Tooth neuropathy
type 2a

Gene: MFN2

Details

We are developing PBCM06 to treat Charcot-Marie-Tooth neuropathy type 2a (CMT2a), a monogenic disease caused by mitofusin (MFN2) gene mutations associated with CMT2a. This neurological disorder presents complex phenotypes, including not only neuropathy-related features but also systemic impairment of the CNS. CMT2a is the most frequent axonal form of CMT, accounting for approximately 20% of the diagnosed cases.

ADULT

Program*
PBFT02

Indication: Frontotemporal
dementia

Gene: GRN

Details

Our gene therapy product candidate, PBFT02, an AAV1 viral vector to deliver a modified DNA encoding the granulin gene (GRN) to a patient’s cells. The vector will be delivered directly to the cerebrospinal fluid by a single injection to the cisterna magna (ICM injection). The goal of this vector and delivery approach is to provide higher-than-normal levels of the progranulin protein (PGRN) to the CNS to overcome the progranulin deficiency in GRN gene mutation carriers.

Related link

Learn more about frontotemporal dementia

Program*
PBAL05

Indication: Amyotrophic
lateral sclerosis

Gene: C9orf72

Details

We are developing PBAL05 to treat amyotrophic lateral sclerosis (ALS), a motor neuron disease characterized by rapid degeneration of upper and lower motor neurons, leading to progressive weakness and premature death. We are targeting ALS patients who have a gain-of-function mutation in the C9orf72 gene. Most cases of ALS are sporadic with an unknown etiology, but there are also genetic forms of the disease inherited in an autosomal dominant fashion. Mutations in the C9orf72 gene are the most common genetic defects implicated in ALS, accounting for approximately 34% of familial ALS cases and approximately 5% of sporadic ALS cases.

Program*
Unamed

Indication: CNS

Gene: Undisclosed

Program*
Unamed

Indication: Alzheimer’s disease

Gene: Target ID

Program*
Unamed

Indication: Temporal lobe epilepsy

Gene: Target ID

*10 additional new pipeline license options.
Program includes ongoing natural history study of infantile and juvenile GM1 gangliosidosis patients.

Passage Bio is conducting three interventional studies to bring potential treatments to patients and families affected by rare CNS disorders

Imagine-1 Logo

We Are Evaluating A New Gene Therapy For Gm1 Gangliosidosis In A Multinational Phase 1/2 Clinical Trial

The Imagine-1 study is evaluating the safety and efficacy of PBGM01, a novel and potentially transformative gene therapy for patients with infantile GM1 gangliosidosis.

Learn more about PBGM01 and the Imagine-1 clinical trial.

GALax-C Logo

We Are Evaluating A New Gene Therapy For Krabbe Disease In A Multinational Phase 1/2 Clinical Trial

The GALax-C study will evaluate the safety and efficacy of PBKR03, a novel and potentially transformative gene therapy for patients with infantile Krabbe disease.

Learn more about PBKR03 and the GALax-C clinical trial.

upliFT-D Logo

We Are Evaluating A New Gene Therapy For Frontotemporal Dementia with granulin mutations In A Multinational Phase 1/2 Clinical Trial

The upliFT-D study will evaluate the safety and efficacy of PBFT02, a novel and potentially transformative gene therapy for patients with FTD with progranulin gene mutations.

Learn more about PBFT02 and the upliFT-D clinical trial.

Father and son lying in bed

In Collaboration With The Orphan Disease Center Of The University Of Pennsylvania, We Are Collecting Information To Better Understand The Natural Course Of Gm1 Gangliosidosis

The natural history study will identify demographic, genetic, environmental, and other factors associated with GM1 gangliosidosis.

Learn more about the natural history study.

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