Six exosome therapy companies driving development in the field

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Exosomes are extracellular vesicles around 30 to 150 nanometers across that are generated by all cells and play crucial roles in fundamental cellular processes, such as the regeneration of damaged tissues. These tiny vesicles are emerging as key tools in personalized and regenerative medicine, offering precise, targeted therapy with low immune risk. With their broad potential for treating a number of diseases, such as cancer, neurological, and inflammatory diseases, several exosome therapy companies have come to the fore in recent times, with many just beginning to advance their candidates through the clinic.

In this article, we take a look at six of the companies leading the way in the exosome therapy field.

Aegle Therapeutics

Positive data reported in phase 1/2 trial for AGLE-102 in second degree-burns
AGLE-102 being investigated in phase 1/2 to treat dystrophic epidermolysis bullosa
AGLE-102 being evaluated in a preclinical program for graft-versus-host disease

Aegle Therapeutics says that its mission is to harness the regenerative, immunomodulatory, and anti-inflammatory potential of mesenchymal stem cells (MSC)-derived extracellular vesicles (EVs), including exosomes, to treat rare and serious dermatological disorders with significant unmet medical need.

In January 2024, Aegle Therapeutics announced positive data for the first patient dosed in a phase 1/2 trial with the company’s lead candidate, AGLE-102 – a therapeutic composite of EVs, including exosomes – for the treatment of severe second-degree burns. Treated with a single topical dose of AGLE-102 within 48 hours of burn injury, the patient achieved 99% epithelialization of the burn wound and had a significant reduction in edema within seven days of treatment.

In addition to the trial for severe second-degree burns, the company also recently started a phase 1/2 trial to investigate AGLE-102 in patients with dystrophic epidermolysis bullosa, a rare pediatric skin blistering disease. Furthermore, AGLE-102 is being evaluated in a preclinical program for graft-versus-host disease (GvHD).

Aruna Bio

AB126 aimed at treating CNS conditions
AB126 demonstrated ability to cross the blood-brain barrier
AB126 set to enter phase 1b/2a clinical trial in acute ischemic stroke

For more than 17 years, Aruna Bio has been leading the research and manufacturing of human neural stem cells in collaboration with some of the world’s leading research organizations.

Now, it is advancing a novel class of cell-free biologics – neural exosomes derived from neural stem cells. The company currently has one lead program, called AB126, to treat some of the most challenging central nervous system (CNS) conditions.

Because AB126 is made up of allogenic neural exosomes derived from neural stem cells, it means that it possesses many of the same proteins as the ‘parent’ cell and is coded for use in CNS applications. Select surface proteins are the key to the blood/brain barrier lock, so it is granted access where other therapeutics are not. This is extremely significant given the challenges in delivering therapeutics past the blood-brain barrier, which acts as a natural protective membrane that protects the CNS from toxins and pathogens in blood.

According to Aruna Bio, preclinical research with AB126 has already demonstrated the ability to cross the blood-brain barrier and concentrate in areas of the brain such as the cerebellum and basal ganglia, which are regions of the brain responsible for motor movements, procedural learning, and cognition.

The program is now set to enter the clinic, as the exosome therapy company announced in January that the U.S. Food and Drug Administration (FDA) had cleared its Investigational New Drug (IND) application for AB126, paving the way for the phase 1b/2a trial in acute ischemic stroke. In one of its more recent announcements, Aruna mentioned that it was on track to initiate the trial in the second half of this year.

Capricor Therapeutics

Lead exosome-based candidate: StealthX vaccine for SARS-CoV-2
Capricor recently partnered with NIH to investigate StealthX vaccine
StealthX vaccine was selected to be part of Project NextGen

Capricor Therapeutics is focused on advancing transformative cell and exosome-based therapeutics, with a mission of redefining the treatment landscape for rare diseases. Its proprietary exosome platform is called StealthX and is for designer protein surface expression, cargo loading, and targeted delivery. The company is currently deploying the technology in two broad modalities: precision therapeutics and vaccinology. Capricor is also internally developing a streamlined manufacturing process to support future development efforts.

Capricor’s lead exosome-based candidate is a multivalent vaccine for the prevention of SARS-CoV-2. The company recently entered into a collaboration with the National Institutes of Health (NIH) to investigate this vaccine. The vaccine was also selected to be part of Project NextGen, an initiative by the U.S. Department of Health and Human Services to advance a pipeline of new, innovative vaccines providing broader and more durable protection for COVID-19.

As part of the project, the National Institute of Allergy and Infectious Diseases (NIAID) – part of the National Institutes of Health – will conduct a phase 1 clinical study with the StealthX vaccine, subject to regulatory approval.

Evox Therapeutics

Evox has two lead assets for CNS indications
Evox partnered with Eli Lilly in 2020 in $1.2 billion deal
Evox raised $95.4 million in 2021

Evox Therapeutics is focused on the treatment of severe rare genetic diseases using their exosome-based therapies. Evox has developed a proprietary technology platform, DeliverEX, to enable it to modify exosomes, load drugs into exosomes, and target the delivery of these exosomes to organs of interest. According to the company, it has successfully demonstrated this capability by delivering drug-loaded exosomes into target organs including the central nervous system and other intractable tissues.

Four years after its inception in 2016, Evox entered into a research collaboration and license agreement worth $1.2 billion with Eli Lilly to leverage Evox’s DeliverEX platform to develop and deliver RNA interference (RNAi) and antisense oligonucleotide drug payloads for the potential treatment of neurological disorders. Shortly after this, the company also raised $95.4 million in series C funding from the likes of OrbiMed and GV (formerly Google Ventures).

Still in the discovery phase, the company is developing a pipeline of drugs leveraging the ability of exosomes to deliver drugs to cells and tissues that are currently inaccessible by conventional means. Its two lead assets are currently for undisclosed CNS indications.

EXO Biologics

EXOB-001 being tested in phase 1/2 to treat bronchopulmonary dysplasia
EXOB-001 granted orphan drug designation by FDA in 2022
EXO Biologics secured $17 million in series A funding in April

With what the company calls a “Triple Axis Strategy,” EXO Biologics is focused on developing exosome therapies for rare inflammatory diseases that currently do not respond to standard-of-care treatments, maximizing the therapeutic potential of exosomes by enabling them to be loaded with specific therapeutic molecules and drugs, and providing high-quality EVs and exosomes from research to clinical use through its GMP manufacturing platform.

Granted orphan designation by the FDA in 2022, EXO Biologics’ lead candidate, called EXOB-001, is being tested to treat bronchopulmonary dysplasia, a severe lung disease that is the most common cause of death in preterm newborns. In June 2024, the company announced that it had completed dosing of the first cohort of patients in its phase 1/2 EVENEW study, which is the first European Medicines Agency (EMA)-authorized clinical trial involving mesenchymal stromal cell (MSC)-based exosomes.

Furthermore, in April 2024, the exosome therapy company announced that it had secured €16 million ($17 million) in series A funding. The company said that the proceeds would go towards supporting its ongoing and future clinical trials, as well as helping it to continue its manufacturing expansion and expanding partnerships to enable pan-market access to its platform and GMP clinical grade exosomes for faster access to clinical trials.

ILIAS Biologics

ILB-202 carries the anti-inflammatory protein super-repressor IκB
ILIAS recently received final clinical study report for ILB-202 from phase 1 clinical trial
No dose-limiting toxicities or serious adverse events at any dose level reported

ILIAS Biologics specializes in developing engineered exosomes by loading large therapeutic payloads inside exosomes via its proprietary platform technology, EXPLOR. The company hopes that by harnessing the power of its engineered exosomes, it can deliver therapeutic proteins to the undruggable intracellular pathways to treat both life-threatening and hard-to-treat diseases.

The company’s lead therapeutic candidate, ILB-202, is an exosome that carries the anti-inflammatory protein super-repressor IκB (srIκB), which inhibits the activity of NF-κB, a pivotal cellular protein that activates inflammation. The srIκB protein prevents NF-κB from entering the cell nucleus and functioning, in turn suppressing the inflammatory response.

In July 2024, ILIAS announced that they had received the final clinical study report for ILB-202 from a phase 1 clinical trial completed in Australia in November 2023. According to the company, this was the world’s first systemic administration clinical trial for engineered exosome therapeutics. The company said that all participants completed the trial without any dropouts, and there were no dose-limiting toxicities or serious adverse events at any dose level. Additionally, no infusion-related reactions or cytokine storms were reported.

Numerous companies involved in exosome-based therapies, as market expected to grow

According to DelveInsight’s exosomes pipeline report, there are now more than 70 companies involved in the field of exosome-based therapies, with more than 80 pipeline therapies for various indications.

Consequently, the global exosome therapeutics market size is estimated to grow by $234.7 million from 2024 to 2028, at a compound annual growth rate (CAGR) of 16.19%. This is primarily due to an increasing prevalence of chronic diseases, with a trend towards advanced technology in exosome collection. However, according to Technavio, strict regulatory approval for exosome therapeutics launches poses a challenge, which is something that companies will need to overcome when they are ready to market their final products.