Target Organ Target
Program Prog.
Indication
(Target)
Ind
Technology Tech
Delivery
Method
Delivery
Discovery Disc.
Lead
Optimization
Lead Opt.
IND-
Enabling
IND
Clinical Clinical
Rights Rights
Target OrganTarget
Program - Indication (Target)
Technology
Delivery
Status
RightsRights
 
 
Arbor
Liver
ABO-101
PH1
(HAO1)
Knockdown+
LNP
ABO-101 - PH1 (HAO1)
Tech: Knockdown+
Delivery: LNP
Status:
Disc.Lead Opt.INDClinical
Arbor Pipeline Logo
ABO-102
ATTR
(TTR)
RT Editing
LNP
ABO-102 - ATTR (TTR)
Tech: RT Editing
Delivery: LNP
Status:
Disc.Lead Opt.INDClinical
Arbor Pipeline Logo
CNS
ABO-201
ALS
(C9ORF72)
Nuclease Excision
AAV
ABO-201 - ALS (C9ORF72)
Tech: Nuclease Excision
Delivery: AAV
Status:
Disc.Lead Opt.INDClinical
Arbor Pipeline Logo
ABO-202
ALS
(STMN2)
Knockdown+
AAV
ABO-202 - ALS (STMN2)
Tech: Knockdown+
Delivery: AAV
Status:
Disc.Lead Opt.INDClinical
Arbor Pipeline Logo
ABO-204
ALS
(SOD1)
Knockdown+
AAV
ABO-204 - ALS (SOD1)
Tech: Knockdown+
Delivery: AAV
Status:
Disc.Lead Opt.INDClinical
Arbor Pipeline Logo
Collaborations
Multiple
Multiple
CF, T1D, next-gen SCD and Beta Thalassemia, other diseases(1)
Multiple
Ex Vivo and
In Vivo
Multiple - CF, T1D, next-gen SCD and Beta Thalassemia, other diseases(1)
Tech: Multiple
Delivery: Ex Vivo and In Vivo
Undisclosed
Vertex Pipeline Logo
Oncology
Multiple
Oncology - Cell Therapy
Multiple
Ex Vivo
Multiple - Oncology - Cell Therapy
Tech: Multiple
Delivery: Ex Vivo
Undisclosed
Edigene Pipeline Logo

(1) CF = cystic fibrosis, T1D = type 1 diabetes, SCD = sickle cell disease

Why Liver & CNS Disease

Since our founding, we have developed a wholly owned portfolio of editing technologies that can be used to create in vivo targeted genetic medicines, beginning with the liver and CNS. Our technology can also be used to enable cell-based therapeutics, and while we are not developing these internally, we have partnered with leading companies who have expertise in ex vivo applications.


The liver has become an important target for genetic medicines, resulting in validated delivery approaches, clear regulatory and clinical paths for products and validated biology—all of which were key factors in our decision to prioritize liver disease as initial clinical indications in which to apply our novel technology. We have selected liver diseases with high unmet medical need where we can provide differentiated therapies for patients. This approach allows us to de-risk elements around our technology for our lead indications.

Our gateway programs are in primary hyperoxaluria (PH) and transthyretin amyloidosis (ATTR). Using proven delivery systems such as lipid nanoparticles (LNPs), we are able to package our editing cargo to deliver to the liver. Arbor’s gene editing approach provides the potential for a one-time dose of disease-modifying therapies that are durable for the duration of a patient’s lifetime.


Beyond the liver, we are also focused on the CNS, where there are many devastating diseases of genetic origin. Until the discovery of gene editing, the technology to effectively target the underlying genetic mutations within these diseases had not existed. This presents an exciting new opportunity for genomic medicine in the CNS. We have selected our initial diseases for targeting in the CNS and our mode of delivery will be via adeno-associated virus or AAV, which is powered to target select regions of the brain that will provide the most meaningful benefit to patients.