VectorY Therapeutics, a biotechnology company pioneering vectorized antibody therapies for neurodegenerative diseases, and Shape Therapeutics Inc. (“ShapeTX”), a leader in applying AI and RNA technologies to engineer next-generation genetic medicines, announced today that they have entered into an option and license agreement. The collaboration is designed to accelerate the development of innovative therapies that leverage deep-brain penetrating adeno-associated virus (AAV) technology for the targeted delivery of vectorized antibodies.
Under the terms of the agreement, VectorY will have an exclusive option to evaluate ShapeTX’s proprietary SHP-DB1 AAV capsid for use with three therapeutic targets. SHP-DB1 is a next-generation AAV5-derived CNS capsid that has demonstrated strong potential to reach previously inaccessible regions of the brain while minimizing off-target risks. Following successful evaluation, VectorY may exercise its option to obtain an exclusive license to SHP-DB1 for delivering vectorized antibodies against these specific targets.
As part of the arrangement, ShapeTX will grant access to SHP-DB1 while VectorY assumes responsibility for advancing development and commercialization of any resulting therapies. ShapeTX will receive an upfront payment and is eligible for success-based milestones and royalties. In total, the agreement could be worth up to $1.2 billion, depending on program outcomes. This includes up to $338 million in regulatory, development, and commercial milestones for rare disease programs, and up to $503.5 million for non-rare disease programs, in addition to tiered royalties on future sales of licensed products.
Strategic Fit for VectorY’s Mission
Jim Scibetta, Chief Executive Officer of VectorY Therapeutics, emphasized how the agreement aligns with the company’s strategy:
“The addition of Shape’s SHP-DB1 technology is a strategic fit with our mission to deliver transformative and disease-modifying therapies for devastating neurodegenerative diseases. Since our founding, we have designated AAV5 as our capsid of choice, and it is already at the core of our lead program VTx-002, a TDP-43 motor neuron–targeting vectorized antibody therapy for ALS. We expect to file IND and CTA applications for VTx-002 by the end of 2025. This new partnership expands our pipeline and enhances our ability to apply AAV5, a clinically validated and safe vector, across multiple therapeutic programs.”
VectorY plans to incorporate SHP-DB1 into several of its ongoing initiatives. Key programs include:
- VTx-003 – a dual-targeting vectorized antibody selective for mutant huntingtin (mHTT) and TDP-43, currently being studied for Huntington’s disease.
- VTx-005 – a vectorized antibody selective for phosphorylated tau, aimed at addressing Alzheimer’s disease.
The ability to deliver these therapies intravenously using an advanced AAV5-based vector has the potential to overcome many of the limitations associated with traditional antibody or gene therapy approaches, offering new hope for patients with neurodegenerative conditions that currently lack disease-modifying treatments.
Why AAV5 and SHP-DB1?
AAV5 has emerged as one of the most attractive serotypes for central nervous system (CNS) applications due to its safety profile and broad CNS tropism. Compared to AAV9, it offers lower hepatotoxicity and reduced immunogenicity, making it a strong foundation for innovative therapies.
ShapeTX’s SHP-DB1 builds on this foundation by providing enhanced deep-brain penetration. Preclinical studies in non-human primates have shown that SHP-DB1 can efficiently deliver genetic payloads to neuronal populations in regions that are largely inaccessible to conventional AAV capsids. Importantly, SHP-DB1 also shows a reduced tendency to accumulate in tissues associated with known AAV toxicities, such as the liver and dorsal root ganglia. This combination of deeper penetration and reduced off-target risk makes SHP-DB1 particularly well suited for the delivery of complex payloads such as vectorized antibodies.
Adrian Briggs, interim Chief Executive Officer and Chief Technology Officer of Shape Therapeutics, expressed optimism about the partnership:
“We are excited to partner with VectorY, whose innovative vectorized antibody approach has the potential to transform the treatment landscape for neurodegenerative diseases. This collaboration showcases the power of Shape’s AAV engineering platform and breakthrough data demonstrating SHP-DB1’s ability to deliver genetic medicines into deep regions of the brain. Together, we can advance therapies that bring new hope to patients and families affected by some of the most devastating diseases.”
Expanding the Therapeutic Horizon
Neurodegenerative diseases such as ALS, Huntington’s, and Alzheimer’s disease remain areas of immense unmet medical need. Traditional small molecules and monoclonal antibodies often face challenges in crossing the blood-brain barrier, while many existing gene therapy vectors lack sufficient penetration into critical brain regions. Vectorized antibodies delivered via advanced AAV capsids present a promising solution, combining the disease-modifying potential of targeted antibodies with the durable expression and delivery efficiency of gene therapy.
By joining forces, VectorY and ShapeTX aim to unlock new therapeutic possibilities that were previously out of reach. VectorY brings expertise in designing and advancing vectorized antibody programs, while ShapeTX contributes its state-of-the-art RNA and AI-driven AAV engineering capabilities. Together, the companies are positioned to push forward a new generation of CNS-targeted therapies designed to address both the root causes and molecular hallmarks of neurodegenerative disease.
Looking Ahead
The option and license agreement reflects a growing trend in the biotech sector, where collaborations between specialized companies enable rapid advancement of transformative technologies. For VectorY, access to SHP-DB1 represents a critical step in broadening its pipeline and validating AAV5 as the vector of choice for vectorized antibody therapies. For ShapeTX, the deal demonstrates the commercial potential of its engineered AAV platform and its ability to generate value through strategic partnerships.
As the collaboration progresses, preclinical data and clinical milestones will determine the trajectory of these programs. If successful, the therapies emerging from this agreement could reshape the treatment landscape for neurodegenerative diseases, providing patients with therapies that are safer, more precise, and more effective than current options.
About VectorY Therapeutics
VectorY Therapeutics is pioneering vectorized antibody therapies to transform the treatment of neurodegenerative diseases. The company’s pipeline is led by VTx-002, a potential best-in-class therapy specifically targeting toxic TDP-43 in ALS, with an investigational new drug (IND) submission planned by the end of 2025 and clinical development planned to start in early 2026. In addition, the company is advancing preclinical vectorized antibody programs including VTx-001 in ALS targeting oxidized phospholipids, VTx-003 in Huntington’s disease, and VTx-005 in Alzheimer’s disease. Backed by leading global life sciences investors, VectorY is advancing a bold mission to deliver one-time, disease-modifying treatments for patients with common but devastating CNS disorders. For more information, see www.vectorytx.com.
About Shape Therapeutics
Shape Therapeutics is leveraging AI to develop new payload, delivery and manufacturing technologies for the gene therapy industry. Alongside the company’s own RNA-targeting gene therapy portfolio, Shape’s platform includes AAV capsids with enhanced tropism and penetration profiles, enabling delivery of genetic medicines to previously inaccessible tissues. The company is headquartered in Seattle, Washington.
