New technologies enable drug delivery to the brain – and open up a billion-dollar market

A smart protection, but also a barrier to treatment

The blood-brain barrier (BBB) is a protective structure that surrounds the brain and only allows passage of certain substances. It plays an important role in preventing harmful toxins, viruses or bacteria from reaching our central nervous system. But the barrier simultaneously blocks out approximately 98% of all small-molecule drugs and almost all biological drugs, including many potentially life-saving treatments for diseases such as Alzheimer’s, Parkinson’s, ALS and brain tumours.

Great medical need and high market potential

The need for effective treatments for central nervous system diseases remains large, with few approved therapies for several serious diagnoses. Forecasts show that the global market for central nervous system treatments is expected to almost double over the next decade – from approximately 130 billion USD today to over 250 billion USD by 2030. Meanwhile, the population over 65 years is growing rapidly, a group where neurological diseases are particularly common.

Technologies that enable effective drug delivery to the brain have the potential to not only improve treatment outcomes but also create entirely new therapeutic opportunities. Today, over 900 pharmaceutical and biotech companies are developing drugs against central nervous system diseases. The field’s global pipeline comprises nearly 1,900 drug candidates, of which over 500 are in clinical phase. Many of these target neurodegenerative diseases, epilepsy and chronic pain – areas where the need for effective drug delivery to the brain is particularly great.

The technologies that can bypass the barrier

Among the most promising methods are:

• Transport molecules: By coupling drugs to proteins that naturally pass the blood-brain barrier, one can utilise the body’s own transport systems. BioArctic’s BrainTransporter technology is an example of this, as are Denali Therapeutics and Roche’s TfR-shuttles (transferrin receptor-based platforms).
• Nanoparticles (biological and synthetic): These are engineered to carry drugs across the barrier and release them in specific parts of the brain. The technology is used for both small molecules and biological drugs. Examples of this include capsid engineering of AAV vectors for CNS-targeting (e.g., Voyager Therapeutics) and synthetic nanoparticles under development by Aliada Therapeutics.
• Intranasal administration: Certain substances can reach the brain via the olfactory nerve and thereby avoid the blood-brain barrier entirely. This non-invasive method is being investigated in pain treatment and in Alzheimer’s disease, among others. Impel Pharmaceuticals has developed an advanced intranasal delivery platform – POD® (Precision Olfactory Delivery) – which enables efficient and targeted administration to the upper part of the nasal cavity, with potential to improve drug delivery to the brain.

Several of these technologies are now in preclinical or clinical phase, and have attracted interest from both venture capital and big pharma.

Increased investment interest and strategic collaborations

Licensing and collaboration agreements in the area have increased significantly, with over 60 transactions in the past five years and a total of 2.5 billion USD in venture capital to BBB technology since 2019.

Interest in BBB technologies is clearly evident in several major deals:

• Swedish BioArctic has in recent years licensed out its BrainTransportertechnology to three global pharmaceutical companies. Most recently in August 2025, the company signed an agreement with Novartis, worth 30 million USD in up-front payment, for the development of a new treatment against neurodegenerative diseases. In 2024, BioArctic also signed a 100 million deal with BMS, and they also have an existing collaboration with Eisai.
• Denali Therapeutics has entered partnerships with Sanofi to develop BBB-penetrating treatments.
• Johnson & Johnson is investing in new platforms that enable CNS access for antibody-based drugs.

What was previously regarded as a nearly impenetrable barrier is now an area where technology is rapidly advancing. Platforms that demonstrate safe and reproducible drug delivery to the brain have the potential to become the next generation of growth companies or attractive acquisition candidates for big pharma.

We expect that BBB-related technology will play a decisive role in the next wave of CNS innovation – and that interest from both investors and pharmaceutical companies will only increase.