Early Translational Research

Editor's Notes

• #2: Thank you for the opportunity to share with you some of my preliminary thoughts and plans in early translational research at UCSF.
• #3: I have put together this framework to help me to simplify and think through the processses in translational research. And this has been useful to me in identifying the key gaps and potential needs. We have what is called early or T1 and later T2 translational research. Under T1, the work of translating science to invention/discovery occurs. Here, I will use the term invention to be synonymous with patent filing while acknowledging that there are discoveries that are not filable which can result in development of products that impact health. Under the T1 umbrella is also where this invention/discovery gets translated to a product that then can be used to impact health and to modify outcomes. I will use the term product to refer to a therapeutic/diagnostic/device/or an entity that can then be used for health benefit. This process is not quite as linear as this graphic would suggest. In an ideal world and in reality, there is feedback from the bedside to bench, from community to bedside and community to bench which informs the science (many would argue we can do better in that regard). In terms of where the resources and focus for the work at the various stages has traditionally been, academia has focused more on the science and discovery. Once there is an invention, start up companies moved the work along towards creating a product. At some point in this continuum, larger companies/pharmas then became involved as strategic partners or through acquisition. I should also mention that large pharma companies also invested significantly into in-house science and discovery work in the past. All analysis would indicate that this investment has been costly and not nearly as productive as hoped for. More recently, esource gaps have been growing. Venture funding for starting up and sustaining biotech/startups is becoming more scarce and the high failure rate of inventions/discoveries in the later phases of development among other reasons have led to large companies/ pharmas being less willing to take on molecules that others have developed at high valuations until substantial development has taken place (reducing the risk that lies ahead). Because internal R& D as well as startups/biotech have become a less reliable source for pipeline, large pharma/companies have been making a concerted effort at reaching directly into academia to get engaged early with the science and to populate, at least in part, their development pipeline in this mechanism. And of course, funding for academic research has been more challenging than ever before. And with the gaps in translational research widening, more is expected with less. In that regard, this new paradigm in which pharma is actively looking to invest in academia and to partner with academia is an opportunity.
• #4: One of the programs T1 traslational catalyst award is a program which was initiated a year and a half ago to address the gap of invention to product. It is intended to identify projects with diagnostic, device, or therapeutic potential The program then provides a customized panel review and in some cases, provided a customized consultation award for the program.
• #5: My thoughts on this early assessment are that the award program only helped to identify a small proportion of the science and invention here at UCSF. And while it’s important to identify the inventions that are ready for support in becoming products, it’s perhaps equally as important to identify the early program to help science to become inventions. And that providing access to the customized consulting and small pilot grants can be a mechanism by which we can identify some of the promising earlier science.
• #9: We are starting the discuss how we might be able to offer the components of T1 Catalyst award thought to be most valuable to more investigators…in particular the pilot grants and the consultation program. Preliminary thoughts around this is that we would create a more comprehensive T1 transalational catalyst program which would include the T1 catalyst award but would also include a freestanding consultation program and pilot-micro grants program for investigators whose programs are less mature and might need a more limited one time consult or a small pilot grant to do that experiment which then can help determine human relevance for the program. Both the consultation and micro grants program would be designed to be minimal process with a quick turn-around on the order of 1-2 wks for decision. Stay tuned for more on this. This can also then be used as a mechanism by which we identify science which have the potential to become inventions. In that way, it can also serve as a funnel to identify programs that can then apply for a full T1 Catalyst Award.
• #13: Early translational research is a complex, multidisciplinary, and in places capital intensive endeavor. The goal is to build innovative partnerships that is fit for purpose in such a way that the scientists at UCSF can continue to do what they do best while leveraging the capabilities that reside in the resources that surround us in the industry and other partners/ collaborators. This might mean that we enter into a collaboration that is more of a co-development model in the way the Pfizer CTI program is set up. This might mean we engage the a venture company to help us get a company started as QB3 has done with great success. We are in active discussions with many of these entities to set up partnerships that are fit for purpose. UCSF/Academia Cutting edge science Deep subject matter expertise and experts Industry partners Development experience Process capabilities Access to resources Potential source for funding