Pipeline

Drug Discovery Strategy

The Tau Consortium strives to support best practice programs with a variety of mechanisms that recognize the distinct challenges of drug discovery and translation for neurodegenerative diseases. Through its vital partnerships, the Tau Consortium’s drug discovery portfolio is supported by integrated expertise and services that cover all aspects of the pipeline from early stage target discovery and validation through clinical trials. Our team works to connect investigators with contract research organizations (CROs), intellectual property consultants, and other necessary services to provide support where it is needed.

With an appreciation for the synergy that results from working collaboratively with like-minded organizations to accelerate the development of therapeutics for neurodegenerative diseases, the Tau Consortium has partnered with the Alzheimer’s Association and The Michael J. Fox Foundation on several initiatives to leverage funding, expertise and outreach. About one half of our drug discovery and translational portfolio is supported through open application initiatives with these nonprofit partners.

Drug Discovery Portfolio

The Tau Consortium funds drug discovery efforts across a wide range of novel targets, agents, and/or therapeutic strategies to accelerate the development of disease modifying interventions for tauopathies. Our pipeline aims to bridge the “valley of death” (the term that scientists use for the time between a basic science discovery and the ability to commit resources to develop that finding into a drug) by cultivating academic labs to grow into more robust translational programs with the ultimate goal of working with pharmaceutical companies to develop their drug candidates for human trials and commercialization.

The funding and discovery strategy is based on the individual needs and stages of the program and can range from assignments of fully integrated drug discovery teams via partnerships and CROs (contract research organizations) and support of drug discovery services via a CRO network with drug discovery and intellectual property strategy advice from experienced consultants. The Rainwater Charitable Foundation stays engaged in supporting and monitoring progress toward proof-of-concept program inflection points where venture financing or other types of funding and pharma partnerships can be realized.

One of the unique qualities of this program is that it encourages the formation of interdisciplinary teams of academic collaborators, contract research organizations (CROs), and/or outside consultants who can assist in moving these projects forward.

Dr. Leticia Toledo-Sherman Senior Director of Drug Discovery

Portfolios

Drug Discovery Partnerships

Tau Pipeline Enabling Program

In 2018, the Tau Consortium and Alzheimer’s Association came together to create the Tau Pipeline Enabling Program (T-PEP). Now entering its third year, the overarching goal of this matching grant program is to accelerate the development of new therapies for tau-based neurodegenerative disorders. T-PEP aims to enrich the “valley of death” drug development pipeline by facilitating innovative but resource-constrained academic projects to become more robust translational programs that pharmaceutical companies may be able to develop into drug candidates for human trials and commercialization.

In the T-PEP I and T-PEP II grant cycles, the partners awarded nearly $7 million to 13 talented research teams. Typically, these two year programs funded through T-PEP are focused on one or more steps in the drug discovery process from target validation and enablement, lead discovery and optimization, proof of concept in vivo efficacy and/or IND-enabling studies. The program is open to researchers at academic institutions as well as small companies.

As co-funders of the grants, the Alzheimer’s Association and Tau Consortium jointly monitor each program’s progress toward project milestones. Continued disbursement of funds is contingent on demonstrated progress toward key milestones.

To read about the winners from the T-PEP I grant cycle, please click on this link.
To read about the winners from the T-PEP II grant cycle, please click on this link.

For more information please visit the T-PEP website here.

PIPETTE Consortium

The PIPETTE Consortium (Philanthropic Investments in PET TracErs) is a funding partnership formed in 2017 by The Michael J. Fox Foundation and Tau Consortium. The purpose of this initiative is to encourage the development of novel 4R tau and alpha-synuclein PET tracers for primary non-Alzheimer’s tauopathies, Parkinson’s disease, Lewy body dementia, frontotemporal dementia, and other neurodegenerative diseases.  As a collaborative effort of leading nonprofit organizations, the PIPETTE Consortium seeks to pool ideas, expertise, and resources to enable the improved diagnosis and treatment of multiple disorders.

PET imaging tools are crucially important to the field. They allow clinicians not only to detect the presence of abnormal protein build-up in the brains of living patients, but also to pinpoint its location and quantify its abundance. PET imaging is expected to be the most accurate way of diagnosing living patients with PSP and other atypical parkinsonian disorders. Taking multiple scans over time will allow doctors and patients to precisely monitor disease progression and treatment response.

Unfortunately, it typically takes several years and millions of dollars to develop a new PET tracer. For-profit companies have little incentive to invest in these tools, so nonprofits must often step in to fill the void. Given the complexity and expense involved, it makes sense for non-profits to pool their ideas, expertise, and resources to ensure success. PIPETTE funds projects with structure- and ligand-based drug design approaches that use state-of-art computational methods based on the high resolution cryo-EM structures of disease-relevant tau and alpha-synuclein fibrils and existing Structure Activity Relationship (SAR) on known tau PET ligands or alpha-synuclein PET ligands that are in development. In addition, the program supports in vivo imaging of brain neurofilaments of 4R tau and alpha-synuclein pathology that could be useful as biomarkers of the presence of disease and disease progression and as pharmacodynamic tools for drug development for tauopathies or synucleinopathies.

In September 2020, the PIPETTE partners launched a new grant program that is open to academic, non-profit, and for-profit applicants. Pre-proposals for this open RFA are due on October 22, 2020. To learn more, please follow this link.

Philanthropic Investments in PET TracErs (PIPETTE)

For more information please visit the PIPETTE website here.

Integrated Drug Discovery Collaborations and Support

Effective drug discovery requires the interdisciplinary expertise of medicinal chemists, biologists, ADME/PK specialists and both in vitro and in vivo pharmacologists to deliver clinical candidates that are progressable through to the clinic. An integrated team approach that can mobilize this collective drug discovery expertise is critical to tackle the complex challenges of optimizing leads into successful candidates for clinical development.

The Tau Consortium supports a drug discovery portfolio of early-stage programs with funding, critical drug discovery expertise, and integrated drug discovery support from CROs (contract research organizations) and partner drug discovery institutes. Teams of drug discovery of experts with diverse backgrounds across multiple therapeutic areas (specifically the central nervous system and neurodegenerative diseases) provide consultation, innovation, and proactive problem solving for the Tau Consortium’s investigators. This expert support is able to guide programs from target validation, hit identification, and hit-to-lead into lead optimization. Our goal is to generate strong proof-of-concept, pre-clinical efficacy data in tauopathy models, supported by pharmacokinetics (the movement of drugs through the body), pharmacodynamics (the body’s biological response to the drugs), and target engagement towards advanced preclinical development and beyond. Our collaborators include the following groups:

• Alborada Drug Discovery Institute: The ALBORADA Drug Discovery Institute is one of three institutes within the Alzheimer’s Research UK Drug Discovery Alliance, as the ALBORADA team works closely with partner institutes at the University of Oxford and University College London. The Alliance will accelerate the discovery of novel, effective therapeutics for Alzheimer’s disease and other neurodegenerative diseases. The ALBORADA Drug Discovery Institute couples the deep disease knowledge and biology expertise of the academic community with high quality, innovative drug discovery technologies.
• The Oxford Drug Discovery Institute: The Oxford Drug Discovery Institute (ODDI) joins high quality drug discovery expertise with a deep scientific and academic understanding of patients, disease mechanisms, and model systems. The intent is to translate cutting- edge academic science into drug discovery and to lead projects through from target to lead status, and beyond.
• Charles River Labs: Charles River provides essential products and services to help pharmaceutical and biotechnology companies, government agencies and leading academic institutions around the globe accelerate their research and drug development efforts. In 2019, the Tau Consortium entered into an agreement with Charles River Labs to provide drug discovery and development support for its investigators.

Outsourced Drug Discovery Services

The Tau Consortium also provides support for outsourcing drug discovery services that include multiple contract research organizations (CROs) accessible via the Science Exchange CRO Marketplace. This partner membership gives access to the TC’s investigators to search more than 2,500 CROs to provide a range of services from early discovery through clinical development. In addition, stand-alone contracts with Charles River Labs and Wuxi extend those services with an extra level of expertise and large array of drug discovery outsourcing services. When necessary, an external consultant can be hired to manage the input and output data or project timeline and costs.

• Science Exchange: Science Exchange is an online marketplace of research service that enable scientists to outsource their research and development to scientific institutions such as university facilities or commercial contract research organizations. The Tau Consortium’s investigators have access to this marketplace of vetted contract services and critical drug discovery expertise to accelerate their research while maintaining best practices.
• Charles River Labs: In addition to providing drug discovery support, Charles River also provides essential products and services to help pharmaceutical and biotechnology companies, government agencies and leading academic institutions all over the world.
• WuXi AppTec: In 2016, the Tau Consortium formed a long-term outsourcing partnership with WuXi AppTec, one of the world’s largest contract research organizations supporting the pharma and biotech industry with drug discovery and development services. Under this arrangement, Tau Consortium investigators can access the specialized services they need to move their work closer to commercialization.

Drug Discovery, Development and Clinical Trials Consultants

The Tau Consortium recognizes the need to couple the scientific insights and inquiry of the investigators with field expertise in drug discovery, development and clinical trials to advance programs towards the clinic. Our team works with a strong group of consulting companies that have experience working on most aspects and stages of drug discovery from early to late stage development.

• Drug Discovery and Development (3D) Consultants: 3D Consultants supports researchers with program-specific scientific consultancy, due diligence and project management. 3D Consultants have expertise across all aspects of the drug discovery and development process with core competencies across a broad range of disciplines including in vitro/in vivo biology, medicinal chemistry, DMPK, pre-clinical development (CMC, tox, scale-up) through clinical development (operations, management, regulatory). In addition, 3D Consultants support our community with preparation of data packages and strategies to access further funding in forms of grants, venture financing and pharma collaborations.
• Audia Consulting, James E. Audia, PhD, provides critical discovery and strategic advice to the Tau Consortium’s staff and principal investigators. Dr. Audia served on the Science Advisory Board of the Tau Consortium from 2014-2020, and he now advises multiple biotech companies and foundations and sits on multiple Science Advisory Boards. He brings a deep knowledge of Alzheimer’s Disease and Central Nervous System Drug Discovery and Medicinal Chemistry. He is the former Executive Director of the Chicago Biomedical Consortium and former Chief Scientific Officer of Constellation Pharmaceuticals, where he continues to serve as a board member. Earlier in his career, Dr. Audia spent more than 20 years at Eli Lilly, where he was named a Distinguished Lilly Scholar (the highest rung on the company’s scientific ladder).

Intellectual Property Consultants

Strategic advice about the protection of intellectual property is a crucial part of the support that our drug discovery investigators receive. Our team works closely with intellectual property experts to help craft program-specific strategies that balance the collaborative nature of the consortium work with necessary protection of drug discovery assets to encourage further investment and partnerships with pharmaceutical companies.

• Global Patent Group. Global Patent Group has critical expertise in intellectual property protection, due diligence and end-to-end patent and portfolio development. This expertise can help guide academic programs with bespoke, program-specific plans.

Drug Discovery Inquiries

The Tau Consortium funds a diverse portfolio of research projects that encourages scientific collaboration to more directly enable academic research, drug discovery and clinical drug development. Our efforts help bridge the translational gap that often exists between academic and industrial research that adds costly delays to therapeutic development. Tau Consortium’s drug discovery portfolio is supported with integrated team expertise and services that cover all aspects of drug discovery and development from early stage target discovery and validation through clinical stages. Our funding assures that general tools, reagents, technologies, assays and platforms developed with Tau Consortium funding can be accessed by any organization or individual needing to accelerate the development of tauopathy therapies. In addition, our funding of natural history studies, clinical patient cohorts, biomarkers and other clinically relevant tools, aims to reduce the barrier to entry into clinical studies for tauopathies.

While participation in the Tau Consortium grant-making programs is by invitation only, we have other funding mechanisms through strategic partnerships and other mechanisms. We would love to learn about your research programs and how we can help enablement. We are happy to answer any questions you have or connect you to additional resources and insights as needed.

Contact our team at drugdiscovery@rainwatercf.org to begin the discussion, or click here to fill out the form.

Thank you for your interest in learning about the Rainwater Foundation’s medical research programs.