VectorY and Shape Therapeutics Forge $1.2B+ Deal for Brain-Penetrant Gene Therapy Delivery
VectorY Therapeutics and Shape Therapeutics have announced a groundbreaking partnership potentially worth over $1.2 billion, aimed at revolutionizing the delivery of gene therapies to the brain. The collaboration centers on Shape's innovative adeno-associated virus (AAV) capsid, SHP-DB1, designed to overcome the limitations of conventional AAV serotypes in accessing deep brain regions.
Strategic Partnership for Neurodegenerative Disease Treatment
Under the terms of the agreement, VectorY will evaluate SHP-DB1's ability to deliver antibody payloads for three therapeutic targets. If successful, VectorY can secure an exclusive license for the capsid technology. The deal structure includes an upfront payment to Shape, with potential milestone payments reaching up to $338 million for rare disease applications and $503.5 million for non-rare disease programs.
VectorY CEO Jim Scibetta emphasized the strategic fit of Shape's technology with the company's mission, stating, "The addition of Shape's SHP-DB1 technology is a strategic fit with our mission to deliver transformative and disease modifying safe and effective therapies for devastating neurodegenerative diseases."
Advancing Neurodegenerative Disease Programs
The collaboration is expected to significantly enhance VectorY's existing pipeline, particularly for intravenous administration of its developmental therapies. Key programs that could benefit include:
- VTx-003: An antibody targeting mutant huntingtin (mHTT) and TDP-43 for Huntington's disease treatment.
- VTx-005: An antibody against phosphorylated tau for Alzheimer's disease.
Additionally, VectorY plans to file an investigational new drug application by the end of 2025 for VTx-002, a TDP-43 motor neuron targeting vectorized antibody for ALS, which utilizes the company's preferred AAV5 capsid.
Engineered Capsids: A New Frontier in Gene Therapy
SHP-DB1 represents a significant advancement in AAV technology. Engineered to penetrate deep into the brain while avoiding liver and dorsal root ganglion toxicities associated with other AAV vectors, it addresses critical safety concerns in gene therapy delivery.
This development comes at a crucial time for the industry, following recent setbacks related to AAV-mediated gene therapies. Notable incidents include two deaths associated with Sarepta's Elevidys due to acute liver failure, and a similar fatality in a clinical trial for an investigational limb-girdle muscular dystrophy gene therapy.
The VectorY-Shape collaboration underscores a growing trend in the pharmaceutical industry towards leveraging AI-driven technologies and engineered delivery systems to enhance the safety and efficacy of gene therapies. Shape Therapeutics has been at the forefront of this movement, having previously secured significant partnerships with Otsuka and Roche, valued at $1.5 billion and over $3 billion, respectively.
References
- VectorY Bets up to $1.2B+ That Shape’s Brain-Penetrant Capsids Are a Fit
VectorY Therapeutics will evaluate the use of SHP-DB1, a capsid developed by Shape Therapeutics, to deliver therapies to the brain, including VectorY’s developmental Huntington’s and Alzheimer’s disease treatments.
- VectorY pens $1.2B biobucks pact to use Shape's capsid to deliver gene therapies into brain
VectorY Therapeutics has seen the shape of things to come—and it involves finding fresh ways to deliver its gene therapies into the brain.
Explore Further
What are the key terms and milestones of the $1.2 billion collaboration between VectorY Therapeutics and Shape Therapeutics?
What is the current competitive landscape for gene therapy delivery technologies targeting neurodegenerative diseases?
How does Shape Therapeutics' SHP-DB1 capsid compare in safety and efficacy to other AAV capsids currently being developed in the industry?
What progress has VectorY made in the clinical development of its pipeline candidates, such as VTx-003 and VTx-005, prior to this partnership?
Are there other ongoing collaborations or similar partnerships in the biotech industry focused on addressing the toxicity challenges in AAV-mediated gene therapies?