A hybrid business model that combines in-house and collaboration programs

In-house pipelines are developed using our expertise in gene modulation by CRISPR-GNDM® technology and transferred through licensing agreements when further development and commercialization is best conducted through a collaboration with experienced partners (❶).
Collaborative pipelines are developed in concert with partner companies, and where CRISPR-GNDM® technology is utilized to advance outside programs (❷).

Revenue Model

The development of a new pharmaceutical product is a time-consuming process that requires significant financial investment before it is commercialized. In the early stages of a business, it is common to raise funds from investors such as venture capitalists (VCs) through the issuance of new shares. However, in many cases, this is not sufficient. Therefore, it is also common to partner with pharmaceutical companies that can provide funding at certain stages of development. In exchange for granting all or part of the sales rights, a one-time payment is received at the time of signing the contract, followed by payments at development milestones as the development progresses, royalties on sales after launch, and sales milestones if specific sales targets are met. This means that revenue can be generated not only after the launch of the product but also from earlier stages.

However, it’s important to note that partnering in the pharmaceutical industry involves a different financial dynamic compared to the sale of general products. The value of the pharmaceutical pipeline increases exponentially as development progresses, so if development is carried out using the company’s own funds and is successful, there is the possibility of obtaining greater future profits. However, there is also the risk of development failure, so the decision will depend on how you think about the balance between risk and return and the funds on hand.

Business Opportunity

It is said that there are around 10,000 human diseases, and of these, there are around 7,000 that are called rare diseases (in Japan, there are less than 50,000 patients, and in the US, there are less than 200,000 patients)

Although the number of patients with each disease is not large, it is said that there are 400 million patients worldwide. More than 80% of these are caused by genetic diseases, and because it is difficult to develop them using the usual pharmaceutical development methods,
there is still no treatment for more than 95% of rare diseases. Against this background, the gene therapy market is growing rapidly worldwide, and it is expected to reach a scale of several billion dollars by 2030.

Our technology is in a position to secure a part of this growing market.

Partner

There is an African proverb that says, “If you want to go fast, go alone. If you want to go far, go together.”
We want to develop the best possible treatment for patients, aiming for the ultimate high. To do this, we are looking for partners who can complement each other’s functions and work together to create better treatments

We specialize in CRISPR-GNDM® technology and are well-versed in epigenome editing, but we do not believe that this alone is sufficient for the development and post-launch follow-up of therapeutic drugs.
Modalis is actively incorporating new technologies and functions to create the best and fastest therapeutic drug development for patients.
We are seeking partnerships with highly motivated pharmaceutical, biotech, and academic companies that have the following technologies and business capabilities.

Technology areas of interest
・ Tissue-oriented delivery technologies, such as capsids for muscles and the central nervous system
・ Technologies to improve selectivity and orientation, such as tissue-specific promoters
・ Technologies to improve protein function
Desired Business Functions
・ Pharma and biotech companies committed to developing treatments for rare diseases
・ Pharma and biotech companies with global development experience and capabilities for drugs for muscles and the central nervous system
・ Companies in the pharmaceutical or other industries that are considering using epigenome editing technology as a tool for drug discovery