VectorY’s VTx-002, featuring an Annogen promoter, cleared for ALS clinical trials phase 1&2

Annogen is proud to announce a major milestone for our partner, VectorY Therapeutics, following the U.S. FDA’s IND clearance to proceed with the PIONEER-ALS Phase 1/2 Trial of VTx-002. This first-in-class vectorized antibody targets pathological TDP-43, the protein driving up to 97% of ALS cases.

VTx-002 aims to provide a durable therapeutic effect by delivering an antibody gene via an AAV5.2 vector, turning the patient’s cells into continuous drug factories. This continuous production is vital for treating progressive diseases like ALS.

The persistent, high-level expression of the VTx-002 antibody within the target cells is powered by a bespoke promoter for which Annogen’s proprietary SuRE™ platform formed the basis for discovery, ensuring essential, long-lasting expression for the genetic payload.

The PIONEER-ALS trial will evaluate the safety and tolerability of VTx-002, which represents a crucial step toward addressing the urgent, unmet medical need in ALS. 

Interested in how Annogen’s promoter technology can optimize your gene therapy platform? Contact us today to discuss partnership opportunities.

 

 

 

 

 

 

 

 

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Annogen Heads to London for the Advanced Therapies Congress 2026

Annogen Heads to London for the Advanced Therapies Congress 2026

Annogen today announced a research collaboration with Orchard Therapeutics, a global gene therapy leader, for the identification of immune cell-specific human promoters for use in certain pre-clinical hematopoietic stem cell (HSC) gene therapy programs.

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