Non-profits wade in where for-profit life science investment fears to tread

For decades, and in the exceptional instance of the Institut Pasteur for over a century, non-profit organizations have augmented R&D to advance scientific understanding and develop affordable and accessible pipelines of vaccines, medicines and diagnostics. Small and medium enterprises and former biopharmaceutical industry executives make a sizeable contribution to global health goals by pursuing opportunities in this corner of the global biopharmaceutical ecosystem.

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At the intersection of biopharma and the public sector sits an extensive international community of non-profit R&D organizations steering life science innovations in the direction of global health burdens (Table 1). Non-profit R&D organizations, not bound to commercial interests, continually cultivate diverse and agile development R&D models to drive product development that saves lives and promotes economic growth. Their diverse approaches to R&D exemplify a far-reaching malleability needed to address major global health challenges like pandemics, HIV, malaria, tuberculosis and neglected diseases disproportionally burdening vulnerable populations. Partnerships and collaborations that include small and medium enterprises (SMEs) and biopharma companies are frequently integral to these endeavors.

Table 1 Key disease indications and the pipelines of organizations leading research and access efforts

Characteristically, the missions and strategies underpinning non-profit R&D organizations work to address technology gaps associated with decades of underinvestment. Funding comes from variety of sources, such as government agencies, foundations, philanthropic and public donors and other organizations. Some organizations draw on royalties and other assets they fostered. Recognition of shared responsibility to address the risk in permitting these gaps to persist drives a creative blend of investment and highly collaborative R&D models. Additionally, non-profit organizations, such as IAVI, Cancer Research UK and PATH, can undertake or mitigate the risk of long-haul challenges such as the development of effective HIV, tuberculosis, cancer or malaria vaccines. For instance, 40 years of research went into introducing two malaria vaccines and advancing HIV vaccine development. PATH’s clinical trial network in sub-Saharan Africa where malaria is endemic was integral to both GlaxoSmithKline and Merck’s ability to obtain regulatory approval in children for their malaria vaccines RTS.S and R21/Matrix-M, respectively. Recently, IAVI, researchers from Scripps Research and others reported phase 1 results that demonstrate proof of concept for their broadly neutralizing antibody approach to immunizing against HIV¹.

Other areas of focus in this socially minded corner of the biopharmaceutical ecosystem include neglected diseases (a group of 21 diseases named for a lack of investment in diseases that disproportionately affect low-income populations), tuberculosis and pandemic preparedness. Impact Global Health is a non-profit that tracks R&D investment trends for infectious, neglected and emerging diseases. Impact Global Health’s Neglected Disease R&D Review reports a 65% increase in products populating the neglected disease pipeline over a ten-year period². Since 2019, the number of drugs and diagnostics in development continued to grow; biologics, microbicides and vector control products stagnated; and vaccines declined. The pipeline is maturing, with 59% of products undergoing clinical trials in 2025. To quantify a state of pandemic preparedness, their 2024 scorecard³ links ongoing R&D to the 100 Days Mission, a research agenda aligning the rapid translation of diagnostics, treatments and vaccines to targeting 12 priority pathogens with pandemic potential. Data in the report show substantial gaps in the R&D needed to prepare for future pandemics.

Places where SMEs step in

Uninhibited by commercial interests, non-profit R&D organizations drive innovation in much the same way companies do. These organizations employ leading experts, translate promising scientific discoveries, cultivate cutting-edge drug discovery platforms, establish international research and clinical trial networks, and augment the pursuit of their mission through partnership, licensing and venture fund investment. The caveat is that the indications peppering the pipelines of these non-profit research-driven organizations and their development approaches do not conform to more traditional investment models that originally cultivated growth of the life science industry, largely across the Northern Hemisphere. Instead, their product development strategies involve de-risking high-risk cutting-edge science. This ensures the affordability and accessibility of products with the added consideration of feasibility factors related to the medicines reaching patients and facilitating a patient’s ability to comply with the treatment.

“One of the key things in looking at the SMEs and small biotechs: they are a huge contributor in terms of the volume of products in the pipeline,” says Nick Chapman, CEO of Impact Global Health. Its G-FINDER database tracks global health investments and pipeline progress toward treatments for neglected and emerging diseases. According to Impact Global Health’s latest data, SMEs topped the list of product contributors for neglected disease indications (Fig. 1). Chapman explains, “The SME sector uses a mix of international grant-based investment from public agencies. At the moment, we are seeing a shift in the US to EU funding. Funding is limited; models have evolved. Reliance on US government funding is changing. There are still investment funds that focus on these diseases, wealthy individuals who contribute, and SMEs across the globe that collaborate.”

Fig. 1: Number of candidates or products by organization type.

Small and medium enterprises (SMEs) contributed the most candidates and products compared to other organization types, according to Impact Global Health’s Infectious Disease R&D Tracker. Combined with academic and other institutions, these two types of organizations contributed just over 60% of the candidates or products addressing neglected and other infectious diseases associated with global health burdens.

“Small biotechs may have the ability to be nimbler and more flexible than other types of organizations or setups. The ability to rapidly adapt and work with us at speed is a vital condition needed to support CEPI’s work and respond to new disease outbreaks,” says Kent Kester, executive director of vaccine R&D, Coalition for Epidemiological Preparedness Innovations (CEPI). Typically, non-profit R&D organizations encompass depth of knowledge, infrastructure and the expertise acquired through years of dedication using donor, public and other funding. These organizations actively open their networks and seek out partnerships and collaborations with organizations that share scientific and/or mission-driven common ground. Kester explains, “We can use CEPI’s existing partnerships with a range of organizations to spot where we could connect SMEs to other sectors to push forward their research.”

Collaboration to the extreme

Characteristically, research-driven non-profits engage in a multitude of collaborations and partnerships to address an unprecedented demand for therapeutics, vaccines and diagnostics. For instance, founded in response to the HIV pandemic in 1996, IAVI is a public–private product development partnership organization. Ayesha Sitlani, associate vice president, product development center, says, “IAVI is poised to translate affordable and accessible candidates across the global health indications in LMICs [low- and middle-income countries].” Sitlani describes a specific opportunity associated with IAVI’s Product Development Center (PDC). “Organizations, governments, industry are able to use our expertise, which goes from preclinical to access, and CMC [chemistry manufacturing and controls]. SMEs who have early assets, that don’t have the know-how, can partner with the PDC.” Similarly, several other non-profits, such as the Drugs for Neglected Diseases Initiative (DNDi), the TB Alliance, Life Arc and Cancer Research UK, have evolved collaboration-rich research translation and clinical trial infrastructures to advance their missions. Former industrial biopharmaceutical executives frequently feature in these communities.

Virtual networks can pave a cost-efficient route to developing affordable medicines. DNDi’s discovery director for drugs, Charles Mowbray, says, “We explore partnership to an extreme.” The 22-year-old organization employs a virtual pharmaceutical development model through collaborations with 200 partners in 50 countries. DNDi’s current pipeline includes 40 discovery projects and about 25 clinical trials.

Initially DNDi picked up abandoned drug candidates and tested repurposed drugs from pharmaceutical company libraries to address neglected disease indications. Fexinidazole, an oral treatment for sleeping sickness, marks the success of this strategic approach. Several countries have eliminated sleeping sickness since DNDi first introduced fexinidazole in 2018. “At a high point, DNDi had access to 12 or 15 major pharma libraries. This dwindled to 3 or 4,” says Mowbray.

“Disengagement by pharma took with it high-throughput screening. To compensate, we looked at alternative ways of working with partners from all sectors. Multidisciplinary teams, small biotechs, universities all have a role to play at different stages in the model we employ,” Mowbray shares. “At the moment, I am excited about our strategy for addressing Dengue.” Dengue is a viral disease, increasing in prevalence as climate change expands the geographical territory covered by the disease’s mosquito vectors. “During COVID-19, we learned it was not easy to use antivirals for acute infections, so we tried to adopt a more flexible strategy that could be adapted to dealing with an outbreak, host-directed therapy.” Host-directed therapy refers to treatments that curtail the human pathology of a disease, an alternative to just killing the virus. “Stop people from needing to go to the hospital,” he says.

DNDI collaborated with BenevolentAI to use the company’s machine learning and artificial intelligence (AI) discovery platform to match pre-existing small molecule therapeutics with “plasma leakage pathways. Leaky vessels can be fatal in the long run; stop progression and you eliminate the need for hospitalization.” After testing a short list of drug candidates in vitro, DNDI is testing a new drug class in animal models.

“An upside is that machine learning can potentially reduce the time to discovery,” says Joan Herbert, executive vice president, business development at Medicines for Malaria Venture (MMV). “The opportunity is in its early days; they [AI and machine learning tech companies] see advantages in working with MMV, as we have a wealth of data generated over 25 years, to train their machines as well as deliver new information to our projects. A synergy exists because our data is open access, and we can share this resource. The early exploratory nature of it makes it exciting.” In March, MMV announced a deal with deepmirror, a machine learning and AI drug design company, to launch a platform named Drug Design for Global Health (DD4GH). Open access and training to use the platform is available to scientists in LMICs working on drug discovery to address serious global health security issues such as malaria, tuberculosis and neglected diseases.

Accessibility, affordability and flexible IP

Non-profit R&D organizations prioritize the accessibility and affordability of products. Approaches to managing and licensing intellectual property (IP) vary in this segment to the biopharmaceutical sector. For instance, in April 2025, the price of pretomanid decreased to the target level of less than $1 per day. Developed by the TB Alliance, pretomanid is used in combination with bedaquiline and linezolid to treat drug-resistant tuberculosis. Pretomanid was the first new drug introduced into a regimen to treat tuberculosis in 40 years. The six-month three-drug ‘BPaL’ regimen shortened the course of highly drug-resistant tuberculosis treatment by over a year and eliminated debilitating side effects associated with the previous standard of care. The Alliance applied a multi-manufacturer strategy to drive down the original price of $364 per course of pretomanid to $169.

“Right at the beginning there is a need to balance accessibility and IP protection particularly,” explains Isabelle Buckle, executive vice-president for technology transfer and industrial partnership at the Institut Pasteur, the first organization to ever undertake research translation for global health. The Institut Pasteur is a private non-profit foundation Buckle describes as “the first startup” and was based on Louis Pasteur’s ability to raise revenue to start an institute with the idea of helping the population through the application of research.

“We outline provisions for licensing to be quick and not delay, and tailor on a case-by-case basis depending on the potential product,” Buckle says. “For instance, it is different for the LMIC or GAVI countries. If licensing a gene therapy for rare disease in children, we would ensure it would be distributed internationally at an affordable price.” Similarly, Kester says, “CEPI’s approach to IP is pragmatic.”

“We require awardees to manage their IP in such a way that equitable access can be realized, including through tech transfers, manufacturing scale-up, affordable pricing and stockpile commitments,” Kester explains. An example is CEPI’s funding for the first-ever licensed chikungunya vaccine, Ixchiq, developed by the French pharmaceutical company Valneva. Valneva’s vaccine is marketed in the United States, Canada, Europe and Brazil. In February, Denmark’s Bavarian Nordic introduced a second chikungunya vaccine in Europe. With support from CEPI and the European Union, Valneva transferred the technology to Brazil’s Instituto Butantan and the Serum Institute of India to produce an affordable supply for LMICs. Kester adds, “These tech transfer partnerships will be key to its local development and rollout.”

At the other end of the spectrum is open science, the primary juxtaposition for DNDi and MMV. “Neglected diseases do not have competitiveness,” says Mowbray, who describes “multiple big companies in the same room talking about their projects. At times it serves as a soundbox for new technology development. Boron chemistry is exciting demonstration of that.” DNDi is advancing a novel boron-based treatment for leishmaniasis and Chagas disease, DNDi-6148, from a collaboration with three companies: Anacor Pharmaceuticals, Scynexis and Sandexis Medicinal Chemistry.

“MMV has some unmovable tenets based on making new medicines affordable and available to people most in need. Nevertheless, whilst adhering to red lines, we always make serious efforts to recognize our partners’ needs in any agreement,” explains Herbert. “In the case of a research collaboration with a university, we recognize the importance to academic establishments of publishing the data arising from the research. We try to accommodate this, when possible, by structuring the legal framework to reflect MMV’s rights to use the data to develop new drug candidates in return for publication rights.”

IP considerations are not the only conduit to facilitating access. In low-income settings, where losing even a day’s work can cause economic hardship, oral formulations make it easier for patients to comply with treatment. For example, working with Sanofi, DNDi introduced fexinidazole, the first all-oral treatment for sleeping sickness — a neglected disease now on track for elimination before 2030. Long-acting injections can bring similar patient benefits. For instance, the TB Alliance is pursuing a regimen-driven R&D strategy to shorten treatment to one month for active tuberculosis and one day to cure latent infection. “To find the right drug with the long-acting technology, we are assessing four or five different technologies and how they stack up with the four or five drugs that we have in the pipeline,” Mel Spigelman, president and CEO of the Global Alliance for TB Drug Development, explains: “Then the patients will not need to take pills every day and will not need to make frequent clinical visits.”

In general, research-driven non-profits provide SME’s and their cutting-edge approaches with opportunities that align with their ambitious goals for increasing affordable access to medicines, vaccine and diagnostics. Chapman explains, “We have seen over the last decade investment in platform technologies,” a trend echoed in the industrial sector. Exemplifying the attraction of SME platform technologies, two relatively young companies, EnsiliTech (Box 1) and ServareGMP (Box 2) demonstrate a private and non-profit approach, respectively. Based on technology platforms developed by the founders, both organizations attract funding and collaborations with multiple non-profits. Chapman adds, “Platform technologies related to process development are strong in [the non-profit] sector, especially lower cost process approaches. Equally, globally, new technology platforms associated with [drug] delivery technologies are real area of promise.”

Box 1 Preventing vaccine waste

EnsiliTech CEO and cofounder Asel Sartbaeva’s original inspiration to introduce thermal stability into vaccines and other biologics came when she observed the doctor take a vaccine from the refrigerator to inoculate her infant daughter. “I was naïve. I wondered why he could not warm it up. I started googling.” After learning that cold chain failures waste at least 25% of vaccines each year, Sartbaeva, who was conducting chemistry research at the University of Bath at the time, asked, “Could my silica-based material help?”

The company’s platform technology, Ensilication, can encase thermally unstable biomolecules within a silica network to prevent degradation at temperatures from –80 to +50 °C. The approach improves shelf life and simplifies transport and storage. After the COVID-19 pandemic revealed difficulties transporting and storing vaccines in many countries, Sartbaeva decided to spin out a company.

From its inception, EnsiliTech attracted a blend of public and private funding. Their venture capital pre-seed round, conducted in 2022, raised $1.5 million from an Innovate UK grant, venture capital firms, a family office and angel investors. Similarly, EnsiliTech’s growing portfolio incorporates commercial and major global health projects improving access to vaccines and biologics in LMICs. “Because we had this well-developed technology from 10 years in the university, as soon as we spun out, we were contacted by a biopharma company, and they became our first collaboration: animal vaccines,” says Sartbaeva. EnsiliTech is currently working with several animal vaccine companies. “They are thinking of commercializing new ensilicated vaccines in 2027,” she adds.

EnsiliTech’s other collaborations include two major UK government R&D projects associated with introducing thermal stability into mRNA vaccines and monoclonal antibody therapeutics. The UK Vaccine Network awarded £2.1 million ($2.5 million) in research funding for EnsiliTech to establish a thermally resilient, ensilicated mRNA–lipid nanoparticle vaccine platform and develop a vaccine for Hantaan virus. Hantaan virus is an emerging disease with pandemic potential. EnsiliTech’s partners on the project include Cape Town, South Africa-based Afrigen Biologics and Vaccines; the mRNA Technology Transfer Programme from the World Health Organization and the UN-backed Medicines Patent Pool; and Houston-based Emervax.

Sartbaeva describes the £1.4 million ($1.8 million) EnsiliTech and the Centre for Process Innovation (CPI) received from Innovate UK in March 2025 as “helping us to de-risk our going into the antibody market.” CPI specializes in prototype development and scaling up for commercial manufacturing. Using trastuzumab as a test case, EnsiliTech and CPI plan to demonstrate the feasibility of integrating Ensilication into a monoclonal antibody production process. Sartbaeva explains, “80% of antibody therapies are used in EU, US and Canada; 85% of the population lives outside those territories. By eliminating the need for expensive and complex cold-chain logistics, we can reduce waste, lower manufacturing costs and expand global access to these life-saving treatments.”

Box 2 Carving a path for affordable biologics

ServareGMP is a non-profit biotech organization founded by Leigh and Gary Pierce, the entrepreneurs who founded PacificGMP, a contract development and manufacturing organization that pioneered single-use manufacturing technology for producing biologics. “As a non-profit formed to deliver biologics medicines currently unavailable in low- and middle-income countries (LMICS), we are applying techniques from for-profit GMP work to reduce costs in production and make much less expensive medicines,” explains Gary Pierce, ServareGMP executive director and co-founder. Leigh Pierce is ServareGMP’s CSO.

“We are a non-profit that recognizes crucial need in the vast majority of LMICs,” Gary Pierce says. He emphasizes, “Someone in Kenya cannot afford a $10,000 drug. Monoclonal antibodies are almost never delivered to middle- to low-income brackets.” As mentioned, the United States, Canada and the European Union consume 80% of all antibody therapeutics. The high cost associated with producing monoclonal antibodies (mAbs) hinders less disparate access. “How do you possibly get monoclonal antibodies to the 7 billion when we are stuck at 100 or 50 dollars a gram for antibodies?” Pierce asks. For a variety of reasons, mAb manufacturing in high-income countries just costs too much to deliver medicines to LMICs.

Reducing the production cost to $10 a gram is the target price set by international non-governmental organizations for making mAbs and other eventually other biologics accessible in LMICs. “Our basic approach is to create highly productive cell, develop high-performance manufacturing processes, and integrate continual manufacturing which all drastically reduce the cost to manufacture mAbs. This is starting to get us to reaching $10 a gram,” Pierce explains.

Grants and contracts from the Wellcome Trust, CEPI and other organizations finance the company’s cholera program and Nipah virus projects, and the organization finances its own continuous manufacturing, disposable-bioprocessing-related R&D. The Wellcome Trust provided early funding to ServareGMP as part of its initiative with IAVI to develop a monoclonal antibody ecosystem in LMICs. ZAC-3, the first biologic to address cholera infections, is ServareGMP’s lead program. ServareGMP routinely collaborates with academic or institutional researchers on early discovery as part of its cost reduction strategy. Researchers at the Wadsworth Center, part of the New York State Department of Health, discovered ZAC-3 (ref. ⁴).

With $43.5 million in funding from CEPI, ServareGMP also leads, in close collaboration with San Diego-based Mapp Biopharmaceutical, the development of an mAb against Nipah virus. Mapp Bio collaborated with Gilead Sciences to develop ZMapp, a combination mAb regimen to treat Ebola virus infection, and nurtures an extensive early-stage pipeline of pathogenic countermeasures. This ServareGMP collaboration is intended to take Mapp Bio’s MBP1F5 beyond phase 1. Later this year, clinical trials will begin in India and Bangladesh, where a reserve of Nipah virus annually causes an outbreak of infections.

Another pillar of the ServareGMP cost-reduction strategy is actively seeding their approach to biologic manufacturing in select LMICs through tech transfer and knowledge sharing. These efforts coincide with the emergence of new manufacturers and organizations such as the Biomanufacturing Industry Development Centre of South Africa’s Council for Strategic Industrial Research, a hub for open innovation supporting SMEs in the country that provides biomanufacturing infrastructure and access to a wide range of bioprocessing expertise.

“We are not restricting our IP,” Pierce states. “The founders agreed we will share with any organization that wants to use what we discover for delivering medicines to those in need. For driving down the cost of goods, sharing is a no-brainer, as long as we get to keep using it. And we will help if we can implement low-cost medicines manufacturing in LMICs. It’s a great way to do what we do, in partnerships around the world.”