Apertura Gene Therapy
Apertura Gene Therapy is pioneering next-generation AAV gene therapies through novel capsid and payload engineering, aiming to overcome limitations in gene delivery and expression. The company is focused on developing genetic medicines by engineering AAV capsids, gene regulatory elements, and payloads to address key challenges in cellular access, gene expression, pre-existing immunity, and manufacturability. Supported by platform technologies from the Broad Institute of MIT and Harvard and Harvard Medical School, Apertura leverages machine learning and high-throughput assays to design effective gene therapies for various indications. The company is actively developing CNS-targeted gene therapy delivery systems, including engineered AAV capsids that can cross the blood-brain barrier via systemic administration, significantly advancing treatments for neurological diseases such as Rett Syndrome and prion disease. Additionally, Apertura is developing CNS and muscle-targeted capsids, such as the CD59 receptor-targeting capsids (CD59 CapXM), which can deliver therapeutic cargo to muscle tissue and the CNS by crossing the blood-brain barrier, promising for neuromuscular diseases.
Industries
Nr. of Employees
small (1-50)
Apertura Gene Therapy
Products
Transferrin receptor-targeted BBB-penetrant AAV capsid (preclinical)
A preclinical AAV capsid engineered to bind the human transferrin receptor to enable intravenous delivery across the blood–brain barrier and achieve widespread central nervous system transduction with reduced peripheral distribution in preclinical studies.
CD59 receptor-targeted capsid for CNS and muscle transduction (preclinical)
A preclinical capsid engineered to bind the CD59 receptor to enable intravenous delivery and transduction of both central nervous system and muscle tissues in preclinical models.
Endothelial-targeting AAV variant (preclinical)
AAV variant developed for high-efficiency transduction of endothelial cells throughout the central nervous system, reported in conference abstracts and poster presentations.
ML-guided capsid screening and selection platform
A platform that integrates generative and predictive machine learning models with high-throughput experimental screening to generate, screen, and prioritize capsid variants for targeted tropism, immune profile, and manufacturability.
Split-intein payload constructs for large-gene delivery (preclinical)
Modular split-intein payload constructs developed to enable functional reconstitution of large genes in target cells, demonstrated in vitro with pathway-specific functional assays.
Transferrin receptor-targeted BBB-penetrant AAV capsid (preclinical)
A preclinical AAV capsid engineered to bind the human transferrin receptor to enable intravenous delivery across the blood–brain barrier and achieve widespread central nervous system transduction with reduced peripheral distribution in preclinical studies.
CD59 receptor-targeted capsid for CNS and muscle transduction (preclinical)
A preclinical capsid engineered to bind the CD59 receptor to enable intravenous delivery and transduction of both central nervous system and muscle tissues in preclinical models.
Endothelial-targeting AAV variant (preclinical)
AAV variant developed for high-efficiency transduction of endothelial cells throughout the central nervous system, reported in conference abstracts and poster presentations.
ML-guided capsid screening and selection platform
A platform that integrates generative and predictive machine learning models with high-throughput experimental screening to generate, screen, and prioritize capsid variants for targeted tropism, immune profile, and manufacturability.
Split-intein payload constructs for large-gene delivery (preclinical)
Modular split-intein payload constructs developed to enable functional reconstitution of large genes in target cells, demonstrated in vitro with pathway-specific functional assays.
Services
Integrated discovery and engineering platform for genetic medicines
A combined computational and experimental platform that integrates predictive machine learning models with high-throughput single-cell and in vivo screening to engineer capsids, regulatory elements, and payloads for optimized delivery, expression control, immune profile, and manufacturability.
Preclinical development and regulatory strategy support
Support for IND-enabling study design, preclinical model selection (including human receptor knock-in models), potency assay development, and regulatory engagement to align on development paths and study plans.
Licensing, sponsored research, and collaboration management
Negotiation and management of licensing and sponsored research agreements and support for sublicensing to enable academic, nonprofit, and industry development programs for receptor-targeted vector technologies and regulatory elements.
Integrated discovery and engineering platform for genetic medicines
A combined computational and experimental platform that integrates predictive machine learning models with high-throughput single-cell and in vivo screening to engineer capsids, regulatory elements, and payloads for optimized delivery, expression control, immune profile, and manufacturability.
Preclinical development and regulatory strategy support
Support for IND-enabling study design, preclinical model selection (including human receptor knock-in models), potency assay development, and regulatory engagement to align on development paths and study plans.
Licensing, sponsored research, and collaboration management
Negotiation and management of licensing and sponsored research agreements and support for sublicensing to enable academic, nonprofit, and industry development programs for receptor-targeted vector technologies and regulatory elements.
Expertise Areas
- AAV capsid engineering for receptor-targeted delivery
- Machine learning-driven sequence and payload design
- Single-cell functional genomics and regulatory element discovery
- Payload engineering for large-gene delivery (split-intein approaches)
Key Technologies
- AAV vector engineering
- Receptor-mediated transcytosis targeting
- Machine learning and generative sequence models
- High-throughput single-cell assays