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  • A decade on from the launch of the pioneering autism biomarker discovery initiative EU-AIMS, here we highlight advances in biomarker discovery and validation from the follow-on AIMS-2-TRIALS programme, which aims to improve the wellbeing and outcomes of autistic people through precision healthcare approaches.

    • Bethany F. M. Oakley
    • Eva Loth
    • Declan G. Murphy
  • INCATE, the INCubator for Antibacterial Therapies in Europe, aims to boost the antibiotic pipeline by accelerating the translation of academic innovation into industrial R&D projects to tackle antimicrobial resistance. Founded in 2021 as a partnership of research institutions and pharma companies, INCATE provides advice, community and non-dilutive funding to start-up companies.

    • Silke Alt
    • Douglas Haggstrom
    • Christoph Dehio
  • Complex innovative designs in clinical trials have the potential to increase efficiency and lower the cost of drug development, improving patient access to therapies. This article highlights designs and approaches based on a meeting linked to an ongoing FDA pilot program in the field.

    • Robert A. Beckman
    • Fanni Natanegara
    • Debra Michaels
  • Pharmacological probes are important tools for exploring disease biology and discovering new therapies. Often molecules of insufficient quality are used instead, leading to spurious and misleading results. The Boehringer Ingelheim open innovation portal addresses this deficiency by sharing extensively validated pharmacological probes with the scientific community.

    • Andreas Gollner
    • Markus Köster
    • Florian Montel
  • Targeted protein degraders (TPDs) are emerging classes of therapeutics that have the potential to address difficult drug targets in new ways. In 2021, experts convened to discuss challenges in TPD safety evaluation, and supported supplementing individual efforts to address these challenges with collective development of novel decision frameworks, tools and a shared evidence base.

    • Lyn H. Jones
    • Constance A. Mitchell
    • Syril Pettit
  • Translation Together, a transnational consortium of translational research organizations, reflects on successes and challenges in regional COVID-19 pandemic responses and proposes five priorities to improve preparedness for future global public health crises and improve the global approach to translational research and science.

    • Kanny K. Wan
    • Danielle Davis
    • Christine M. Cutillo
  • The International Rare Diseases Research Consortium (IRDiRC) is a global collaborative initiative launched in 2011, aimed at tackling rare diseases through research. Here, we summarize IRDiRC’s vision and goals and highlight achievements and prospects after its first decade.

    • Lucia Monaco
    • Galliano Zanello
    • David A. Pearce
  • The European Lead Factory combines assets and experience from major pharma with innovation and agility of academia and SMEs in a collaborative platform to expand access to high-throughput screening. With many successes heading towards the clinic, the organization is broadening its approach to screening and partnering.

    • Philip S. Jones
    • Sylviane Boucharens
    • Jon S. B. de Vlieger
  • In 2020, the UK government funded a portfolio of platform trials to develop new treatments for COVID-19. A key feature was the independent prioritization of candidate drugs with central coordination to prevent duplication, accelerating recruitment to deliver definitive trial results. A similar approach could be used for non-communicable diseases where treatment advances have been limited.

    • Patrick F. Chinnery
    • Marion Bonnet
    • Tao You
  • An increasing number of genome-editing technologies are being developed. Here, we highlight some of the key issues for future therapies using these technologies from a regulatory perspective.

    • Philip A. Hines
    • Eleonora Agricola
    • Ralf Herold
  • Repurposing off-patent drugs has long been proposed as a cost-effective and efficient strategy to develop new treatments, but its potential has not been realized. Here, we highlight some of the barriers that underlie this lack of progress and discuss potential strategies to address them.

    • Christopher P. Austin
    • Bobbie Ann Mount
    • Christine M. Colvis
  • ENABLE is an antibacterial drug discovery and development consortium formed as a public–private partnership in 2014 as part of the Innovative Medicines Initiative (IMI) New Drugs for Bad Bugs (ND4BB) programme. With the project soon ending, here we provide a brief overview and reflect on its achievements, strengths and weaknesses.

    • Marie Olliver
    • Laura Griestop
    • Anders Karlén
  • Ten years ago, GlaxoSmithKline established an innovative model for public–private research partnerships in Latin America known as Trust in Science. Here, we summarize this novel initiative, illustrate its achievements in terms of scientific advances and local talent development, and highlight the key learnings that could be applied to similar initiatives.

    • Israel S. Gloger
    • Rosana Felice
    • Kevin P. Madauss
  • Misconceptions about translation — defined as the process of turning observations in the laboratory, clinic and community into interventions that improve the health of individuals — hinder efforts to close gaps and address challenges related to the translational process. This article highlights some misconceptions with the aim of improving understanding and advancing solutions.

    • Christopher P. Austin
  • Providing support for medicine development by academics and clinical researchers is a priority for the European Medicines Agency. The recent launch of free scientific advice to academia and non-profit research organizations developing orphan medicines can help align research with regulatory requirements early during medicine development and enhance the chances of success.

    • Rosa Gonzalez-Quevedo
    • Constantinos Ziogas
    • Anthony Humphreys
  • Greater investment is needed in antibiotic R&D, and more must be done to maximize the impact of such investments. More widespread data sharing, such as the recent joint data contribution from Merck and Kyorin to the Pew Charitable Trusts’ SPARK platform, has a key role.

    • Wes Kim
    • Kevin Krause
    • Kevin Outterson