50 Best Companies to Watch 2020

A pioneer in drug delivery with an innovative targeting technology: VECT-HORUS


Many diseases around the world still remain without suitable treatment options. This unfulfilled need for treatment is a major incentive in research and development of efficient cures. For example, many central nervous system (CNS) disorders, such as Alzheimer’s and Parkinson’s disease are becoming more prevalent. This is putting increasing pressure on pharmaceutical companies to produce more effective drugs for brain disorders. Many potential leads are developed. However, they are limited by their inability to pass from blood vessels to brain tissue. Indeed, the vascular system of the brain, known as the blood-brain barrier (BBB), is endowed with unique features. It is a highly selective interface that excludes from the brain virtually all large molecules (notably biologics) and more than 98% of all small-molecule drugs generated by chemistry. Therefore, technologies that enhance drug delivery to the brain are of great interest to pharmaceutical and biotechnology companies, and the potential market for these technologies is growing.

It is in this context that VECT-HORUS designs and develops vectors that facilitate the delivery of drugs or imaging agents into organs, notably into the brain, and also to tumors. The biotechnology company, based in Marseille, France, was founded in 2005. VECT-HORUS has developed its VECTRans® technology, unique in Europe, for the development of molecular “vectors” that facilitate the transport of drugs or imaging/diagnostic agents to the brain and other tissues (e.g., cancers).

In conversation with Alexandre TOKAY, Co-Founder, CEO and Chairman of VECT-HORUS

Q. How does the therapy work for CNS and cancer?

The vectors we develop target specific receptors (receptor-mediated transport or RMT) that are present at the BBB and are naturally involved in the transport of nutrients for the brain. BBB receptors capture their natural ligands in the blood compartment as well as our vectors conjugated to drugs or imaging agents and shuttle them across the BBB into the nervous tissue. Similarly, cancer cells are endowed with receptors or cell surface markers that are not found in healthy cells; our vectors conjugated to imaging agents or anti-cancer drugs allow targeting of these cancer-specific receptors, resulting in increased uptake of the conjugates into cancer cells. The objective is to focus the treatment on the diseased cells while sparing healthy cells and organs to reduce toxicity and side effects. The Company has already demonstrated proof of concept of its technology in animal models by conjugating various drugs to its vectors.

Q. How do you overcome the limitations and drawbacks that are present in targeted therapies?

Targeted therapies benefit from immense progress in biology and chemistry in the last 20 years, and from the increasing potential to conjugate chemical and biological molecules. These therapies will undoubtedly expand with our increasing knowledge on the expression of specific receptors or markers at the surface of cells and the state-of-the-art technologies implemented to develop specific vector molecules, most of them based on biologics (peptides, protein ligands, antibodies or fragments thereof). Such vectors can now be directly conjugated to numerous organic or biologic drugs or can functionalize different types of nanoparticles, including liposomes that can encompass a variety of drugs. The RMT technology is weaving its way from preclinical investigation to clinical use.

Q. Tell us about your patents and their contribution to your success.

Vect-Horus has secured its technology with several families of patents issued in various countries, including the USA, Europe, etc. These patents cover its libraries of vectors and also vector drug conjugates developed in house for cancer imaging and treatment. They allow the Company to implement its business model based on: i) research collaborations and licensing agreements with industrial partners to target their drugs to the brain and other organs; ii) building a pipeline of proprietary drug candidates with the aim of out-licensing them to Pharma or Biotech companies following preclinical or early clinical studies. Currently, the Company collaborates with more than ten different Pharma/Biotech R&D companies for CNS and cancer indications. For these collaborations, Vect-Horus evaluates a variety of therapeutic agents which are unable to cross the BBB effectively or to reach cancer tissue, including proteins, nucleic acids, antibodies, nanoparticles, etc. Agreements with some of these partners contain provisions for a steady flow of capital via upfront and milestone payments, which we will use for future internal development programs.

Q. What are the future plans for the development of your company?

Vect-Horus is leveraging its technology platform to build a pipeline of drug conjugates, targeting indications with strong unmet medical needs such as CNS diseases and cancers. The drug candidates are developed/co-developed either as proprietary drug candidates or in collaboration with pharma/biotech companies. The CNS market is likely to grow significantly in the next few years since a number of pharmacological compounds are being developed for the treatment of CNS-related diseases. In the field of cancer, there is also an urgent need for early and accurate diagnostic methods, coupled with better therapies. Clearly, in the next few years, Vect-Horus is ideally positioned to participate actively in developing solutions for drug delivery to the brain, cancer, and other indications. The R&D collaborations signed with the different Pharma/Biotech Companies position Vect-Horus as a major player worldwide in the field of RMT technology.

Meet the leaders behind the success of VECT-HORUS


The VECT-HORUS was co-founded by Alexandre Tokay, CEO and Chairman, and Dr. Michel Khrestchatisky, Scientific Counsel. Management also includes Dr. Jamal Temsamani, Director of Drug Development & Corporate. Vect-Horus is a spin-off of the Institute for Neurophysiopathology (INP) directed by Dr. Michel Khrestchatisky and supported by the CNRS and Aix- Marseille University. VECT-HORUS currently has 35 employees, most of whom are scientists. Since its inception, VECT-HORUS has raised about €30 million of which €20 million from private investors and €10 million through different types of public funds.

“Vect-Horus’ core expertise relies on modifying pharmacokinetic (PK) and distribution profile of therapeutic or imaging candidates.”