While there are many reasons why a promising discovery fails to translate into a treatment, in Australia, there is a distinct phenomenon whereby projects are unable to reach a stage where they are attractive for investment by pharmaceutical companies, venture capitalists, government or philanthropic organisations. It’s possible for research on a potential target to be published in a high-impact journal but not generate any interest for further commercialisation from the broader sector. The BioCurate team brings funding, industry expertise and hands-on management to help scientists translate their promising biomedical discoveries into high quality preclinical candidates to accelerate commercial outcomes. 

Why does this gap exist, what is the experimental data needed to bridge this gap and how exactly can BioCurate help?  

 

What makes a project attractive for investment?

Projects need to be attractive to investors. The process of bringing a novel treatment to regulatory review and approval can take more than 10 years and over USD $200 million (using the example of development of a small molecule in oncology). With such financial risk, only the most promising projects with the greatest commercial opportunity are considered for investment. Therapeutic candidates need to be supported by rigorous, reproducible and robust scientific evidence. That is, there needs to be a convincing case as to why a novel therapeutic candidate should be further developed. 

Projects are attractive for investment when they address: 

• Unmet medical need 
• Clear commercial and licensing potential
• Differentiation (preferably on efficacy) from other approaches on the market, or in clinical development, or from those that have failed in development  
• Verified link to human disease – target validation  
• Available and predictive biomarkers 
• Intellectual property (IP)
  • Must have potential for novel IP. Existing IP is not necessary
  • No known Freedom to Operate (FTO) issues
• Clearly defined regulatory and clinical pathway
• Must be supported by robust, reproducible scientific data

 

What is the “gap” and why does it exist?

The therapeutic development pipeline can be segmented into a number of stages. While most modalities will go through similar stages, the processes involved at each stage may vary slightly. The following is the typical pathway in the development of a small molecule drug: 

1. Basic Research & Target Identification (ID) 
2. Target Discovery 
3. Lead Identification 
4. Lead Optimisation
5. Preclinical Development 
6. Clinical Trials 
7. Regulatory Review & Approval 

Often, biomedical research that takes place in academic settings ends at the Basic Research & Target ID stage. Here, a target of interest (gene or protein) with a potential role in disease is identified through data mining (bioinformatics), genetic association studies, expression profile experiments, pathway and phenotypic analysis or functional screening. 

Industry, however, rarely invests in projects at this stage as it is deemed “too risky.” An analysis by BioCurate has found that the majority of deals are done at the “pre-clinical” stage, not Target Discovery, and that return on investment is highest when exiting at the Lead Optimisation stage (Figure 1). Here, risk has been mitigated to an acceptable level whereby industry is prepared to invest the millions necessary to further develop a candidate.  

 

As such, there is an expertise gap between where academic research ends and where industry begins (Figure 2). This gap commonly covers the Discovery Phase which includes Target Discovery, Lead Identification and Lead Optimisation activities. This means that promising projects that have produced high-impact publications may be left languishing with their potential unfulfilled.  

 

 

How to close the Gap

To close this gap, academic scientists need to undertake further research activities to reach the Lead Optimisation stage. With this data at hand, industry is more likely to invest with the aim of putting a novel therapeutic candidate on market. But what does this entail?

1. Target Discovery and Validation 

At this stage, academic researchers need to experimentally demonstrate that a target is directly involved in a disease process, and that modulation is likely to have therapeutic efficacy. Experiments/tools may include: 

• Expression analysis
• Mechanism of Action studies
• In vitro function assays
• In vivo validation experiments
• Bioinformatics

2. Lead Identification or Hit-to-Lead

Here, “hits” from screening campaigns are transformed to “leads” through refinement of compound potency and selectivity. This can include activities such as: 

• High-throughput assay development
• Hit identification
• Secondary and orthogonal screening
• Hit-to-lead development
• Structure-based drug design
• Structure activity relationship studies
• Target engagement studies

3. Lead Optimisation

This stage involves the refinement of lead compounds, culminating in the identification of a preclinical candidate demonstrating on-target activity and in vivo efficacy. This may involve:

• Lead characterisation
• Structure activity relationship studies
• Optimising lead potency, efficacy & specificity
• ADME (absorption, distribution, metabolism and excretion) assays
• Target engagement studies
• PK (pharmacokinetic)/PD (pharmacodynamic) efficacy studies
• Pre-GLP (good laboratory practices) toxicology studies

 

Why is it so difficult to close the Gap?

Closing the gap is difficult because of the breadth and depth of expertise needed to undertake, analyse and understand the data generated in the Target Discovery, Lead Identification and Lead Optimisation stages. For example, with the development of a small molecule, these three stages alone may require a team consisting of: 

• Project leader
• Computational chemist
• Medicinal chemist
• Biochemist
• Molecular biologist
• ADME specialist
• Toxicologist
• Pharmacologist 

Regardless of the modality, a multidisciplinary diverse team with specialised expertise is critical in providing all the skills and knowledge needed to progress a project. Academic researchers cannot do it alone. BioCurate is here to help close the Gap.  

 

BioCurate’s role 

BioCurate’s team of experts bring hands-on industry experience and a global track-record in the successful development and commercialisation of a range of therapeutic modalities spanning a diverse array of diseases.  

BioCurate is unique in that not only does the company provide funding opportunities, but the BioCurate team works collaboratively with academic researchers in a hands-on manner to conduct discovery activities. As well as BioCurate’s licensed portfolio of projects that receive funding and expertise to reach the “exit” stage of investment by Venture Capital or large Pharma after Lead Optimisation, BioCurate provides support for early-stage projects so that they can achieve the first steps described above. By funding early Proof of Concept studies and then actively driving the translation of projects utilising the team’s expertise in rigorous biopharma business practices, BioCurate is there for the long haul, as opposed to many other funding bodies. 

BioCurate is committed to supporting and mentoring talented academic researchers to help translate their projects, and to ultimately fulfill our joint vision of delivering clinical impact to patients through the development of new therapies. 

Return to Insight

This tension often leads to the misconception that academic scientists must choose one or the other.

Fortunately, publication and commercialisation are not mutually exclusive and, when planned carefully, will be advantageous for both the academic and commercial partner. There is a way to safely continue publishing papers while progressing a project along the development pipeline with a well-considered publication strategy.

 

What is considered public disclosure and how does it affect commercialisation?

The forms of “public disclosure” can include publications as well as abstracts, posters, and oral presentations at conferences and meetings. This also includes presentations within your own institution.

Any public disclosure of your work by you, or others, may be used by patent examiners as examples of “prior art.” Prior art is any information (art) that may demonstrate that the perceived novelty in potential intellectual property (IP) does not actually exist, as that information was publicly available prior to an application for legal protection of the potential IP.

While public disclosures may vary in their extent, timing and audience, if they are not carefully and strategically planned, they risk the future of a promising potential therapeutic.

 

What is a publication strategy?

A good project will achieve both publication and commercialisation. When a project starts, a publication strategy should be developed in conjunction with the timeline. With careful planning, publications can add value to commercial deals.

Exploratory biology publications are good as they attract interest to the area. However, it is key to maintain one’s competitive advantage (e.g., by avoiding premature disclosure of a validated target (or means of targeting) to maintain one’s developmental lead time) so that you preserve the possibility of being first to market and still attract investors further down the development pipeline by offering them something of value that they themselves have not yet explored.

Publications around development of the novel therapeutic itself need to be carefully staged. For example, if you are developing a small molecule drug, it is desirable to create more than one chemical series. One series could be developed as a “tool compound” which can be disclosed and published. The other series (or multiple series) would be developed as the potential drug candidate and advanced along the drug discovery pipeline with no external disclosures (see Figure 1).

 

 

A similar approach applies to other therapeutics such as antibodies. Antibody sequences we well as their mechanism of action (if unique) should not be disclosed and/or published until an appropriate value inflection point (see Figure 2). This applies to both therapeutic antibodies and ‘tool’ antibodies such as ‘surrogates’.

 

 

When to Apply for a Patent

Timing of patent filing is crucial. Only patent when you have something of true value to investors. Filing for a patent too early will reduce the time window for recouping costs and providing a return on investment. As such, in order to attract the large amount of industry investment required to take a candidate drug through clinical trials, sufficient patent life must remain at the end of that process.

To get a patent granted, you need to be able to make novel and non-obvious claims, and a strong patent position is essential to attract investors. Patents with broad unexemplified claims will incur significant costs and time as they will almost definitely be subject to rejections once they reach national phase.

You also need to ensure that no earlier patent (including your own!) will count as prior art against you.  Remember that as soon as a patent publishes (18 months after the earliest priority date), everything contained within is public knowledge. Should you decide your invention needs more work your patent will be cited by examiners as prior art against your new claims.

 

Different Types of Patents

There is no such thing as a novel target. The genomes of most species have been sequenced, and all potential targets are public knowledge. It is the application of the target to a disease process, uncovering its function, and means of therapeutic use which is valuable IP.

If a target has been linked to a novel biology, a ‘methods of use’ patent application could be filed. This will protect the new biological concept, link to disease, and therapeutic mechanism of action. A ‘methods of use’ patent could apply to using existing reagents for a new purpose (e.g., use of existing monoclonal antibody for a companion diagnostic).

Even if there is no linkage of a target to a disease process or novel function, but a new therapeutic (and variants thereof) is being developed, a ‘composition of matter’ patent application should be filed. This protects the novel sequence of your therapeutic antibody or the inventive medicinal chemistry steps to make a therapeutic small molecule and will claim some of the characteristics of the molecule (i.e., potency, cross-reactivity, and/or developability). A composition of matter patent for a final therapeutic is typically filed after a ‘methods of use’ patent and, since it defines and protects your actual drug candidate, is the basis of the drug’s potential value. Thus, protecting this IP and filing it as late as possible is preferred.

 

How can BioCurate help?

BioCurate is committed to supporting researchers in achieving impact from their research and advancing their career through publications. This commitment, together with extensive knowledge and experience  working at the intersection of research and industry, enables the BioCurate team to develop a publication strategy that can result in desired academic and commercial outcomes.

Return to Insight

Having completed her PhD in immuno-oncology at the Peter MacCallum Cancer Centre, Jaclyn moved to Boston for her postdoctoral studies. Being immersed in the city, she saw the opportunities in industry- how it opened doors to other prospects and aspects of biotechnology, how it would give her a better understanding of drug discovery, and how it could help her make an impact on clinical practice and the lives of patients.

“Academia often paints industry in a negative light, but you learn so much in industry. You learn about what makes a good drug or drug target outside of just compelling biology, which is knowledge you would rarely be exposed to in academia,” explained Jaclyn.

Moving to Seres Therapeutics and then Bristol Myers Squibb (BMS), Jaclyn not only learned about what makes a drug possible but also the many stages a promising candidate could fail. Here, she worked on projects covering a broad range of modalities and therapeutics, from microbiome therapeutics to protein degraders to small molecule inhibitors, while deepening her knowledge of the specificities of each modality and her understanding of key concepts such as pharmacodynamics and pharmacokinetics.

As part of her role as Principal Scientist at BMS, Jaclyn scanned internal screens, projects from collaborations with academic scientists and the literature for potential drug targets. From there, she wrote proposals as to the viability of the drug target, analysing its biology, chemistry and the competitive landscape for the target. For targets that progressed, then began the experiments. Jaclyn managed a multi-disciplinary team of biologists, chemists, toxicologists, and in vivo pharmacologists to help meet the milestones for proof of concept, hit identification, hit-to-lead, lead nomination, and in vivo studies, after which the project would have been handed to the clinical team ready for Investigational New Drug Application (IND) filing with the Food and Drug Administration (FDA). “Over my time at both Seres and BMS, seeing a promising drug target progress through towards clinical trials, was enormously gratifying,” said Jaclyn. “I’m excited to do the very same thing at BioCurate alongside researchers around Melbourne.”

The ease with which she switched and jumped between projects, Jaclyn credits to her time in academia. Jaclyn commented, “The experience of a PhD and postdoctoral research helped me gain a broader understanding of different types of biology. In industry, especially in a large biotech company, you need to be able to adapt to new projects outside your field of expertise, read the literature, and then digest that information. It was easier and faster when you already had a solid foundation in the biology.” She also believes the soft skills scientists develop in academia are often underappreciated. The postdoctoral experience of being tasked to independently drive research was critical in her professional development, as was the time spent on crafting abstracts, talks and grant proposals. “Writing a proposal for a potential drug target, you have to understand…why should I or anyone else care? Which is certainly a question I had to answer many times to other scientists and grant panels during my time in academia and a skill I took into industry.”

Her best piece of advice for academic scientists is to understand that “just because a target isn’t a good drug candidate, doesn’t mean the research isn’t informative.” Having seen a fair few projects meet an early demise, Jaclyn has learnt the importance of letting go. However, she understands how difficult this may be. “Many academics have built their careers, their livelihoods on one or a handful of genes or proteins, so there can be a strong emotional attachment. But in industry, if a project doesn’t meet a milestone, it gets dropped. There is a high rate of attrition on early drug discovery programs, and this is because the bar is very high to ensure only the projects with the best chance of success make it through.” It is a matter of reframing, Jaclyn believes. “Negative” data is still a part of the data package that forms around a drug target and helps inform future projects of any potential pitfalls, adding to the wealth of information and knowledge that moves the drug discovery process forward.

“And when a project ends, it is only ending now. It’s not necessarily forever. Look at protein degraders which have the potential to target previously “undruggable” targets through a mechanism of action only recently identified. We are only now realising the potential, when it wasn’t possible before. Technology moves swiftly and has made these types of drugs possible. It’s all about being resilient while knowing when to pivot.”

Return to Insight

Eric joined BioCurate in late 2017, teaming up with Dr. Cathy Drinkwater to seed the development of the BioCurate project portfolio from initial interaction with academic researchers to project selection and through to oversight and delivery of specific project milestones. From the onset, he worked closely with inventors and liaised with the Universities and other key players in the commercialisation pipeline.

As a classically trained pharmacologist with over 25 years of scientific experience as an academic, a commercial scientist, an entrepreneur and a consultant, Eric is well versed in traversing the many stages involved in drug development and relishes the dynamic challenge of “staying across the breadth and depth of disruption and convergence of technology that is shaping new therapeutics development.”

While Eric began his career by undertaking a PhD in medical pharmacology at the National University of Singapore and then completing his postdoctoral studies at Monash University, his interest in science was sparked when he was a 12 year old track and field athlete suffering from the adverse effects of anti-histamines. He reflected, “Trying to run a sub-28 minute 8K, being on anti-histamines and left sedated with no muscle tone. There had to be better drugs.”

As part of his quest to find better drugs, Eric moved into industry where he was in two different teams that delivered compounds into the clinic for cardiac and respiratory indications, which he lists as some of his happiest career achievements thus far. Others on this list include his time in Indonesia, where he worked with colleagues to establish a contract research business that has improved the lives of young students and scientists from that country in particular, and more broadly across the Asia-Pacific region.

Eric’s passion for the ultimate goal of improving the lives of patients, empathy for researchers trying to make important discoveries under difficult circumstances and understanding what investors and potential partners need to get discoveries to patients aligns perfectly with BioCurate’s values. “The team that Glenn, Linda and Damien have assembled is awesome, the mission and vehicle are unique and the potential to have impact across the spectrum from researcher to investor to patient is inspiring.”

Return to Insight

For academic researchers in the early stages of drug discovery, understanding the ethos, the attitudes and processes that comprise industry best practice is an important prerequisite for embarking on the commercialisation journey. Here, we explore what comprises industry best practice, principles of engagement between parties during drug development, and how you can optimise your chances of successfully bridging academia and industry.

This document will aid researchers in familiarising themselves with the expectations of collaborating with industry to translate early-stage research.

Communication and Collaboration

It can be understandably challenging for researchers who have invested many years, even decades, to gain a deep understanding in a particular field, to then feel comfortable handing on that research to others. However, the successful translation of great ideas into real-world impact requires input from multiple disciplines. One of the first major differences that may be noted is that an industry research Project Team does not only consist of research scientists. Depending on the stage of development, the team could include project managers, research scientists, toxicologists, process development and technology transfer specialists, quality experts, manufacturers, clinical operations staff, bio-statisticians, regulatory affairs experts, clinicians, finance and marketing specialists, legal and intellectual property (IP) experts.

The successful scientific entrepreneur must be willing to work with individuals who have expertise in different fields and to engage in robust debate about data – not only with respect to the science, but also with regards to validation of their results, assumptions about clinical use, differentiation from competitors and market potential. Sometimes, this can be confronting, but as we all know deep down, skepticism and constructive criticism is a critical pillar of research and key to a healthy, collaborative partnership.

Open, clear and honest communication also extends to matters of intellectual property, presentations and publications. Disclosing results in public forums must be managed strategically. Implicit in this strategy is consideration of costs and benefits of disclosure, prioritisation and management of tradeoffs from both an academic and industry perspective. For example, investors may not be willing to invest if disclosures have the potential to compromise intellectual property undermining the commercial endeavour. Once a publication strategy has been agreed, a detailed publication plan can be created, i.e. a roadmap detailing what to present, when, and in what forum, be it seminars, conferences, or a publication. It is important to stress that industry involvement does not preclude publication. Industry will often want to publish research alongside their academic counterparts, however, timing and content may differ and will be agreed as part of the Publication Plan.

Communication and collaboration are critical to the commercialisation process. To effectively drive and manage projects, we recommend you work with a partner who can bring commercial insight based on extensive industry experience and provide you with honest feedback made in good faith, working closely with you to translate new discoveries into new medicines.

Raw data and Validation

The conversation surrounding the ‘reproducibility crisis’ has demonstrated the clear need to thoroughly and completely ensure the robustness of results. For industry, standard practice involves constantly and consistently validating data, even when statistically significant results have been presented from earlier experiments. The emphasis here is on the validation of reagents and repeating experiments, ensuring systematic use of positive and negative controls, blinding, randomisation, benchmarking, a priori definition of endpoints and success, adequate powering and confirming results with an orthogonal method. In addition, raw data will often be independently re-analysed.

This is not a sign of distrust or doubt in the original research but is simply, due diligence. It is a sad truth that many independent studies have found the reproducibility of scientific findings to be poor, with one stating that no more than 25% of assessed published preclinical studies could be validated to the point at which projects could continue.

Considering the time and cost of developing potential drug candidates, a thorough vetting process is critical and as such, is the norm in industry. With a robust evidence base that supports the development of a drug candidate, there is less risk and thus, greater confidence. Ensuring that your data is “robust” and reproducible will greatly assist in the odds of securing a deal with a biopharmaceutical company to translate a discovery made at the lab bench into a therapy used at the bedside.

Funding and Timelines

Funding allocation in industry is specific and focused, which often differs from how the more familiar funding bodies from government or philanthropic organisations operate. Industry funding is usually aligned with a tightly prescribed set of activities, deliverables and milestones, with a strict requirement for adherence to budgets and timelines. Funds are often staged or tranched, meaning a milestone or Go/No Go point needs to be achieved in order to trigger release of the next tranche of funding.

As well as timelines and the meeting of experimental milestones that are consistent with industry standards, the successful execution of a drug development program relies upon regular, responsive and dynamic communication between all members of the research and project teams. Collaborators must be accessible and do their utmost to avoid any delays in response times. If delays in communication or collaboration become systematic and unresolved, milestones may not be reached on time leading to possible project termination.

While all BioCurate funding is necessarily directed towards the activities detailed in the overall project plan, the specifics will vary depending on the project. We focus on optimising the three pillars of execution: timeline, budget and quality. In some cases, we will outsource research to a CRO who is accustomed to high throughput or undertaking research activities at scale. In other cases, we prefer to work directly with research labs at the University given their unique expertise and availability of specific models. In these situations, funding may be used to support extra personnel in the University researcher’s laboratory.

Working with you

Our team has expertise in both academia and industry and have been directly involved in multiple partnerships, in bringing drugs to market in several different modalities and therapeutic areas all over the world. Experience from the early-stage discovery process through to approval from the U.S. Food and Drug Administration (FDA), European Medicines Agency (EMA) and the Pharmaceuticals and Medical Devices Agency (PMDA) in Japan has informed our team of the processes, knowledge, behaviours and mindset that are required to see a drug candidate all the way through the development and commercialisation pipeline.

Ultimately, BioCurate is here to discuss the potential of your research project and to ready scientists like you for the next stage of an exciting journey that may see your research translated into products that improve the quality of patient’s lives. There is nothing more exciting, rewarding or valuable than seeing that vision realised!

Return to Insight

The notion of “Success” is relative and highly contextual. This is true of biomedical research and development. Depending on where you are in terms of the therapeutics development pipeline, both organisationally and functionally, you are likely to get a different definition of success.

For the academic researcher undertaking important wet lab research, who makes that initial breakthrough discovery, success is typically linked to the publication of a manuscript. Success is the novel, landmark or first-in-man finding; it is the paper that is accepted into a high-impact journal (Cell, Nature or Science- the holy trinity of biomedicine publishing). Publication in these high-impact journals is an accepted surrogate for the importance of the initial discovery, and the potential for changing research paradigms and benefiting patients. This achievement is appropriately seen as success: it is an important first-step along the way to something valuable for patients. As a consequence,  it also increases the chances of future funding, promotion, and international recognition.

For the entrepreneur, success is seeing a market opportunity and bringing capital and technology (often the technology is based on the breakthrough discovery in the academic research laboratory) together to address it. Through this process the entrepreneur creates value and value creation is the yardstick by which their success is measured. For the investor, success is the identification of opportunities (which are often generated by the entrepreneur) to deploy capital and achieve the greatest financial return. This may be achieved by securing an exit with an industry partner. These contributions can be considered a second-step along the pathway to ultimate patient benefit.

But for the biopharmaceutical industry, success is a viable commercial product on the market. As such, it can be seen the final step on the pathway to bringing benefit to patients. It is seeing drug development through to the very end, with new treatments in the hands of doctors and patients and seeing the many years of research finally make a positive impact on human health and quality of life. Within the company each function that contributes to the overall company goal has different definitions of success – for manufacturing it may be reducing the cost of producing the new drug (“cost of goods”), for clinical operations it may be efficient enrollment of patients in the clinical studies, for medical affairs it is ensuring patient safety, for regulatory it is expeditious approval by the Regulatory Authorities, and for the health economics and outcomes research team it is achieving reimbursement for the new therapeutic approach.

For the patient and society in general, success is an efficient use of taxpayer dollars that funds relevant research that can be efficiently transferred to the entrepreneur who brings it together with investor capital to create further value through an exit to an industry partner. Ultimately, this results in affordable access to cost-effective treatment that improve outcomes for all who need it.

Misalignment across players can cause delays, disputes over the allocation of resources, counterproductive competitive behavior, and can compromise the commercial viability of projects entirely. Our definition of success needs to be realigned to one that is mindful of the end goal – to see a novel treatment on the market and available to patients.

In this context, success for the academic, the entrepreneur, the investor and industry are but milestones in the continuum, each worthy of celebration – crucial interdependent steps towards societal success rather than ends in themselves.

For promising new projects, creating opportunities for effective partnering of all of these players is a fundamental premise for the development of novel therapeutics. In these partnerships, misalignment in the different parties’ understanding of success or a “zero sum game” mentality can be problematic. Our societal mission of bringing safe and effective therapeutics to patients does not stop with the success of one player in the development chain. Misalignment across players can cause delays, disputes over the allocation of resources, counterproductive competitive behavior, and can compromise the commercial viability of projects entirely.

Our definition of success needs to be realigned to one that is mindful of the end goal – to see a novel treatment on the market and available to patients.

This is why BioCurate exists. BioCurate is doing its part to bridge the gap between academia and industry by pursuing opportunities to align success metrics. BioCurate exists to further develop, for patient benefit, the outstanding fundamental research for which Australia is famous. It is acknowledged that there are systemic pressures that influence the way research is conducted in Australia. Changes to precarious funding models and the insidious pressure to publish are necessary. A more long-term, commercial mindset is critical to help our country realise the significant potential of its research community and achieve success in commercialising promising new discoveries.

BioCurate is striving to improve our collective understanding of the realities of translation, and to bring industry to researchers- to facilitate new collaborations. Creating a strong, multidisciplinary team of experts and supporting the entire drug development value chain will fortify the foundations for success and improve the likelihood of bringing novel treatments to market that improve the lives of patients.

Return to Insight

“As with most research institutes, the Monash BDI is making a concerted push to improve industry engagement amongst academics. I was acutely aware of BioCurate, as a few groups within the Monash BDI had already been interacting with members of their team,” said Roger.

As discussions evolved between Roger and BioCurate, a mutual understanding developed – that the rigour applied to interpreting certain types of data, even by scientific journals, is insufficient. Roger recalled, “The team at BioCurate were responsive, they saw the worth of our work and also thought our results were interesting.” However, a major problem was identified by both Roger and the BioCurate team – that the potential off-target effects of siRNAs are often underestimated.

“Glenn (Begley, CEO of BioCurate) and I agree- the standards applied when using siRNAs are not stringent enough. There are certain kinds of controls which are recommended, but to be honest, very few people actually take on these controls to the extent they’re supposed to. This is really where the problem resides,” said Roger. “Many journals simply request that the effect is replicated by at least two siRNAs. However, the effects of both can be off-target.”

Based on these discussions, Roger and his team are undertaking further experimental work using alternative approaches to strengthen their case, and this is already reaping dividends. “We’re keeping BioCurate informed as this validation work progresses, because the data are not only critical to our project, they have more general relevance to how BioCurate, and industry, assesses projects,” he said. This is what has pleasantly surprised Roger – the ongoing conversation that has developed following that initial presentation.

For Roger, this process has been and continues to be a positive and highly encouraging experience, and one that has galvanised his opinion on the best approach to translational research. “Labs going into translational research need to go in with eyes wide open, they need to be aware of potential problems as well as options and opportunities,” he explains. “If you want to complete a drug screen, it is important to ask- are we best placed to do it? Or can we outsource it?” Roger wants scientists to leverage the funding, skills, expertise and resources available to them, rather than ‘going it alone from scratch.’ This may prevent groups from taking on work that doesn’t match their skill base, and that may divert resources away from their strengths.

“Melbourne has outstanding facilities and a fantastic research community,” stated Roger “BioCurate is well-positioned to advise on how to tap into such resources and move projects down the development pipeline.” He added, “I’m looking forward to the prospect of this project reaching the point at which it’s ready to enter the BioCurate pipeline.”

All in all, it’s all about the big C – Collaboration – to help achieve real world impact.

Return to Insight

Helping this big picture come together is the big vision that BioCurate has, and it was what drew Cathy to the organisation. ‘I completely agree with Glenn [Dr C. Glenn Begley, CEO of BioCurate]. Australia is too small to compete on the global stage as individual parties. We need to work together.’ This emphasis on collaboration and cooperation was born from Cathy’s experiences during her PhD.

For Cathy, undertaking her PhD at the Howard Florey Institute was one of the best times of her life. ‘Of course, there was plenty of frustration, but by the time I was writing my thesis, I just enjoyed the feeling of having achieved a certain depth in my knowledge and the sense of completing something. I had a lot of autonomy and I revelled in being my own boss.’ For her, what was underrated about the entire PhD experience was the personal development that went beyond the confines of the lab. ‘You learn to become a team player, whether it’s negotiating equipment bookings or learning how to deal with different personalities. These skills are so valuable.’

After postdoctoral positions at Stanford University of School of Medicine and the Walter and Eliza Hall Institute of Medical Research, Cathy made the transition into industry. Beginning as a Principal Scientist at AMRAD Corporation (later Zenyth Therapeutics), Cathy was still actively involved in research. ‘However, in industry, it is research with a purpose. A purpose to develop new drugs,’ commented Cathy.

One of the starkest differences between academia and industry that Cathy noted was the motivation that drove research. ‘ It’s push vs pull: in industry, you know where you want to go – your work is being pulled through and guided by the end goal. In academia, new discoveries can push your research in different directions, sometimes with a lack of focus.’ It was also in industry where Cathy began to understand the importance of robust and reproducible research  data. ‘There is a necessary ruthlessness in industry. If we couldn’t replicate the data, we dropped the project. It just wasn’t worth pursuing.’ This focus on reproducibility is one that is very much at the forefront at BioCurate.

After her time as Project Manager at CSL, managing projects at various stages along the development pathway, Cathy moved back to early discovery projects at Cancer Therapeutics CRC, which she now continues at BioCurate. It’s a perfect fit for her. ‘I love being exposed to exciting new discoveries, although the slower pace of academic research can sometimes be a bit frustrating,’ she says. Despite this, Cathy is pleased to see scientists from both academia and industry beginning to act on the need for collaboration. Cathy comments, ‘Seeing multiple groups working on the same type of projects, but not talking to each other didn’t make sense to me. I’m so glad that it’s definitely changing now.’

Her extensive and expansive experience, as well as her expertise in endocrinology, neurobiology, oncology, immunology and inflammation, positions Cathy as an authority on early drug discovery, but also a leader in fostering stronger ties between the seemingly disparate worlds of academia and industry. She understands the needs and nuances of both.

For scientists who want to bridge this gap and to contribute to the development of novel treatments, she offers the following advice: ‘Be open to collaboration. Find people you can trust and who can offer you new ways of looking at your scientific problem. All my best successes have involved multidisciplinary teams.’

Return to Insight

‘I think academic researchers are mindful of the fact that they need to translate their discoveries. But how to actually go about doing that remains somewhat of a black box,’ says Dr Natalie Borg. This sentiment is shared by many biomedical researchers and reflects the knowledge and experience gap that exists between a basic discovery and translation of research into something that has real impact.

In the academic environment, because of the lack of expertise and funding, this gap is known as “the valley of death.” Yet this valley is routinely traversed within industry. It is therefore essential to demystify this process, and provide the resources and know-how that exists within industry to bridge this gap if we are to realise the full potential of academic scientific discoveries.

The challenge, however, facing the translation of academic science to achieve impact for human health is demonstrated by the increasing cost of drug development over recent years. According to the Tufts Center for the Study of Drug Development, during the 1990s, the development of a new drug took an average of 8 years with costs up to $USD802 million. Today, that figure stands at 15 years and $USD2.6 billion, in spite of dramatic advancements in knowledge and technology. This quantifiable inflation in cost and time has driven a greater outward research focus of industry as it seeks to harness academic advances. BioCurate was specifically created to foster a stronger understanding and collaboration between academia and industry – to bridge the academic valley of death.

Professor Dale Godfrey and Dr Natalie Borg, two researchers who have maintained long-running conversations about their respective projects with the BioCurate team, understand this problem all too well. Professor Dale Godfrey is currently an NHMRC SPRF in the Department of Microbiology and Immunology at the University of Melbourne and Immunology Theme Leader at the Peter Doherty Institute for Infection and Immunity; and Dr Natalie Borg is an ARC Future Fellow and lab head based in the Department of Biochemistry and Molecular Biology at Monash University. While each are at different stages of their career and commercialisation journeys, both have come to realise they no longer need to view their research and achieving its commercial potential as separate endeavours.

With the uncertain path to commercialisation and the publish or perish mentality firmly entrenched in academia, “research-translation” can simply become an empty cliché. Both Dale and Natalie have had indirect interactions with the world of commercialisation throughout their academic careers. Dale completed his postdoctoral research in the United States, in an academic environment that was funded by industry. He recalls, ‘there was a friendly lawyer who would wander around the labs, asking about any new findings that could be patentable.’ He has also consulted for and embarked on small collaborations with several companies. Natalie undertook her PhD in a lab and at an institute that was heavily involved in the development of the influenza drug, Relenza®. This exposed her to the research path that led to commercialisation. ‘Through absorption, I learnt a lot and found the process quite inspiring,’ says Natalie.

In spite of this early exposure, for both Dale and Natalie, working in the highly competitive research grant-funded academic environment in Australia meant that the need to present and publish research has taken priority over advancing research in collaboration with industry. Combined with limited information and few examples, it has been difficult for researchers to seriously contemplate commercialisation. Buzzwords such as commercialise and translate are often bandied about, but tangible means to progress such aspirations are limited. Today, advice on how to pursue this pathway is increasingly accessible and opportunities are becoming more apparent, sparking a shift in the mindset of many academic scientists.

BioCurate was designed to help facilitate this change in attitudes through education and true partnership, allowing academic scientists to explore the potential avenues to effect impact on human health. ‘It just wasn’t standard thinking. When faced with exciting results, I would think about how good it will be to present them at an upcoming conference, how to turn them into a publication or how I’ll use them for a grant application,’ states Dale. Things are beginning to change. ‘Now I’m also thinking about whether new results we have in the lab might have commercial and clinical potential by way of industry engagement.’ The guidance BioCurate has provided to Dale has given him a clearer view of the industrial therapeutic landscape, the opportunities and options therein, and the potential courses of action. It has helped him understand the best way to navigate sensitive areas of confidentiality and how this path can be managed in the context of other academic pressures.

For Natalie, who co-leads one of the six drug discovery projects that were selected for BioCurate’s first round of project investments, the application process was, in and of itself, enlightening. ‘It was the first time I really had to think about my research assets, who am I competing with, what is on the market?’ said Natalie. Currently, BioCurate’s support has mobilised her science, providing her the resources to push forward, as well as advice on finding the balance between publishing data and maintaining the commercial viability of her research. Not only that, but Natalie has found the chance to tap into the expertise of the BioCurate team to be hugely beneficial, educational – and enjoyable! Through this, she is learning a new vocabulary – one that is aiding her in conversing with industry. Her deeper understanding of the complexities and nuances of commercialisation is facilitating this journey.

BioCurate operates in the under-resourced yet highly critical early phases of new drug development. This phase represents an unmissable opportunity to grow greater links and collaboration between academia and industry and by doing so, will improve the development of potential treatments, for small molecules and biologics, across different therapeutic areas. BioCurate bridges this gap by providing the critical ‘missing link’ – industry experience and clear commercial focus; targeted funding and project management; mentoring and support – that will enable researchers to maximise the impact of their research.

Return to Insight