Pipeline

New hope for people with previously untreatable central nervous system disorders

Our gene therapy portfolio spans pediatric and adult CNS indications. We are advancing lead clinical programs in GM1 gangliosidosis and frontotemporal dementia (FTD) and have built a promising preclinical pipeline including candidates for amyotrophic lateral sclerosis (ALS) and Huntington’s disease.

A FOCUSED PIPELINE OF PROMISING GENE THERAPIES

Program

Indication

US/EU prevalence

Discovery
Pre-clinical
Phase 1/2
Pivotal

Program
PBFT02

Indication: Frontotemporal
dementia - GRN

US/EU prevalence: 18,000

Details

Our genotype therapy product candidate, PBFT02, an AAV1 viral vector to deliver a modified DNA encoding the granulin genotype (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 genotype mutation carriers.

Related link

Learn more about frontotemporal dementia

Program
PBFT02

Indication: Frontotemporal
dementia - C9orf72

US/EU prevalence: 21,000

Program
PBFT02

Indication: Amyotrophic
lateral sclerosis

US/EU prevalence: 72,600

Program
PBFT02

Indication: Alzheimer’s
disease - rs5848 SNP

US/EU prevalence: 3.9M

Program
Unnamed

Indication: Huntington’s Disease

US/EU prevalence: 60,000

Program*
PBGM01

Indication: GM1
gangliosidosis

Genotype:

Program*
PBKR03

Indication: Krabbe disease

Genotype:

Program*
PBML04

Indication: Metachromatic
leukodystrophy

Genotype: