Our Platform
Built to advance genetic medicines.
From discovery through clinical development.
Aurelix brings scientific innovation and integrated therapeutic development together to build genetic medicines for rare and ultra-rare diseases.
The Development Platform
Scientific depth. Connected execution.
Our platform connects the scientific and development disciplines needed to build and advance complex genetic medicines.
Discovery & Translational Research
Target identification, therapeutic strategy, and translational evidence to guide development decisions.
Nonclinical Development
Integrated pharmacology, biodistribution, toxicology, and safety strategies to support clinical advancement.
Manufacturing & CMC Strategy
Product development and manufacturing strategies aligned with the therapy, regulatory requirements, and long-term development needs.
Regulatory Development
Regulatory strategy, agency engagement, and submission development across the therapeutic lifecycle.
Clinical Development
Patient-informed trial design, meaningful endpoints, medical leadership, and clinical execution.
Program Leadership & Execution
Integrated program planning, innovative development strategies, and sustained program stewardship to optimize timelines, resources, and development costs across the therapeutic development journey.
We design therapeutic programs with speed, efficiency, and cost in mind from the outset. By aligning nonclinical, CMC, regulatory, and clinical development, leveraging existing knowledge and infrastructure where appropriate, and reducing unnecessary duplication and rework, Aurelix creates more efficient pathways toward clinical investigation and continued development.
Scientific Expertise
Different modalities. Development built around the science.
Our experience spans multiple genetic medicine approaches, with scientific and development strategies tailored to the biology, therapeutic modality, and needs of each program.
AAV & Lentiviral Gene Therapy
Gene replacement and other vector-based therapeutic approaches, with strategies tailored to disease biology, delivery, and development requirements.
Genome Editing
Therapeutic genome-editing approaches, including precision editing and base-editing strategies for rare genetic diseases.
RNA Therapeutics
Antisense oligonucleotides (ASOs), siRNA, mRNA, and emerging RNA-based therapeutic and delivery approaches.
Emerging Modalities
Exploring novel therapeutic and delivery approaches that may create new opportunities for rare and ultra-rare disease treatment.
Personalized & Individualized Therapeutics
When the patient defines the program.
Some rare diseases affect so few patients that a traditional development model is not feasible. Aurelix has built the scientific, regulatory, and operational capabilities to advance individualized therapeutic programs—where a single patient or a small group of patients defines the development program.
Therapeutic Areas
Where the science leads.
Our programs span a range of rare and ultra-rare diseases, with a focus on conditions where genetic medicines offer meaningful therapeutic potential.
Neurological & Neurodevelopmental Diseases
Metabolic & Lysosomal Storage Diseases
Rare Oncology
Rare Cardiovascular Diseases
Designed for Continued Development
Built to go further.From first patient to global approval.
Every development decision at Aurelix is made with the full arc of a program in mind—from the first patient dosed through continued development toward global regulatory approval.
We build programs designed to succeed not just at the next milestone, but at every milestone that follows.
Build With Aurelix
Let's build the bridge from science to patients.
Together, we can turn promising science into a coordinated development program with a path toward clinical investigation and continued progress.