Pipeline
6 Assets
Patients
1,247
Clinical Development Timeline
Therapeutic Pipeline
Six Programs Across Rare Disease & Oncology
Phase II
HLX-204
AAV9 / Spinal Muscular Atrophy
Single-dose intrathecal gene replacement targeting SMN1 restoration in Type II/III SMA patients.
Enrolled
312
Phase II
HLX-118
LNP-mRNA / Hepatocellular Carcinoma
Lipid nanoparticle delivery of tumor suppressor mRNA with immune checkpoint co-stimulation.
Enrolled
187
Phase I
HLX-371
AAVrh10 / Duchenne Muscular Dystrophy
Micro-dystrophin gene transfer via novel capsid with enhanced muscle tropism and reduced immunogenicity.
Enrolled
84
Preclinical
HLX-509
CRISPR-Cas13 / Alpha-1 Antitrypsin
RNA-targeting CRISPR approach for liver-directed correction of SERPINA1 misfolding without permanent DNA edits.
IND Target
Q3 '26
Preclinical
HLX-622
AAV-DJ / Retinitis Pigmentosa
Subretinal delivery of optogenetic actuators restoring light sensitivity in rod-cone dystrophy models.
IND Target
Q1 '27
IND Filed
HLX-088
LNP-siRNA / ATTR Cardiomyopathy
Second-generation siRNA therapeutic with extended hepatocyte durability and quarterly dosing schedule.
FDA Review
Active
Scientific Advisory Board
World-Class Expertise in Gene Therapy
JW
Dr. James Wilson
Pioneer in AAV vector biology and gene transfer
UPenn Gene Therapy
LN
Dr. Lena Nilsson
Structural biologist, CRISPR delivery systems
Karolinska Institute
RK
Dr. Raj Bhatt
Former CSO, led 3 gene therapy programs to BLA
Ex-Spark Therapeutics
MC
Dr. Maria Chen
Oncology gene therapy, LNP platform design
MD Anderson