Round up: Cracking the hidden code of the human genome through AI, and more

Age Tech World explores the latest research developments in the world of longevity and ageing.

Pushing the frontiers of generative AI for longevity

AI- driven drug discovery and development company Insilico Medicine is launching an innovative cardiometabolic disease portfolio of unique highly-differentiated molecules discovered using generative AI.

Powered by Insilico’s proprietary end-to-end Pharma.AI platform, the portfolio covers a range of diverse mechanisms and stages from early discovery to preclinical development.

It consists of eight oral small molecules with unique properties designed to unlock the full potential of established high-confidence targets such as GLP-1R, GIPR, Amylin, APJ, and Lp(a), as well as moderate-novelty targets such as NLRP3 and NR3C1.

The two novel GLP-1RAs are designed for improved safety and pharmacokinetics at low dose to allow for multi-pill combinations with the other molecules in the portfolio. One of the GLP-1RAs is designed to sustain once-weekly (QW) dosing.

“Cardiometabolic and antiobesity drugs such as GLP-1RAs may be the first wave of longevity therapeutics increasing both healthspan and lifespan in a large population” said Alex Zhavoronkov, PhD, founder and CEO of Inislico Medicine.

“With our cardiometabolic strategy we decided to choose several established and moderate-novelty targets but deliver a high level of differentiation through novelty in chemistry focusing on properties to help unlock the full potential of the targets in a variety of diseases and biologic processes and allow for combinations at low dose.

“Our multi-parameter optimised GLP-1RAs are oral small molecules with very high levels of preclinical safety tested in multiple species and unique molecular structure that can potentially sustain once a week (QW) dosing.”

The introduction of Insilico’s new cardiometabolic portfolio, powered by its Pharma.AI platform, features eight programs targeting seven distinct targets, spanning from lead optimisation to IND-enabling stages.

At the center of the portfolio’s lineup are two oral small-molecule candidate agonists targeting the GLP-1 receptor (GLP-1RAs), which have reached preclinical candidate (PCC) stage and form the basis of the portfolio’s metabolic effects designed for mono- and combination therapy with the other molecules.

Also nearing the PCC stage, a small molecule targeting NR3C1 is designed to treat hypercortisolism-associated metabolic diseases.

In the early stages of the R&D pipeline, four additional programmes targeting GIPR, Amylin, APJ, and Lp(a) are currently in the lead optimisation phase. AI-driven development has led to significant differentiation and enhanced properties compared to existing therapies.

Collaboration to crack the hidden code of the human genome through AI

ARC Innovation at Sheba Medical Center and Icahn School of Medicine at Mount Sinai are entering a landmark three-year collaboration with NVIDIA to harness the power of AI for genomic discovery through the application of large language model (LLM) technology.

The initiative aims to pioneer the decoding of the majority of the human genome that remains poorly understood in order to unlock new pathways for disease prevention, diagnosis, and treatment.

Over the three-year period, the project will be supported by significant investment from the participating organisations, with the goal of accelerating research that can ultimately benefit patients worldwide.

The collaboration brings together three global leaders across health care, technology, and research: Sheba and Icahn School of Medicine will contribute extensive genomic datasets, AI research capabilities, and clinical insight, while NVIDIA will provide its advanced computational architecture and AI development platforms, software, and scientific expertise.

Together, the institutions will create an AI-driven genomic research engine capable of identifying patterns and regulatory mechanisms that link genetic variation to disease risk and therapeutic response.

“This collaboration is an important step toward a future where every person can benefit from the power of whole genome sequencing,” said Alexander Charney, director of the Charles Bronfman Institute for Personalized Medicine.

“By bringing advanced AI into genomic research, we’re moving closer to making personalised, precision medicine a reality for all.”

The initiative’s initial focus will be on areas of medicine where genetic complexity has long hindered scientific progress.

The platform will analyse the interplay of thousands of genomic regions to help researchers identify underlying mechanisms that contribute to disease and to uncover potential therapeutic targets.

Joint research teams from all three institutions have already begun laying the foundations for a shared platform that will support scientists and clinicians worldwide.

US$4m gift to advance women’s longevity

Linda Moslow and husband, Jeff Moslow, chair of the Tufts University Board of Trustees, have donated US$4m to help shape a Women’s Health and Menopause Initiative.

The donation marks a first-of-its-kind academic collaboration uniting the leading expertise of Tufts University’s schools of medicine and nutrition with the renowned Tufts Medicine health system.

The Moslows’ gift endows two inaugural professorships – one at the School of Medicine and one at the Friedman School – whose holders will co-direct the initiative and drive its interdisciplinary agenda.

Under the co-directors’ leadership, the initiative will advance clinical care, education, and research in novel ways that improve women’s health across the lifespan.

The initiative presents a model for how universities can mobilise their full range of expertise to address women’s needs.

In addition to advancing research, clinical practice, and education, Tufts seeks to elevate the national conversation by placing women’s health at the center of academic inquiry and public discourse.

The investment is essential, says Linda Moslow, especially in light of how one landmark study upended the course of women’s health care.

Two decades ago, a large federal study – the Women’s Health Initiative – changed the field almost overnight when its early findings linked hormone replacement therapy (HRT) to increased risks of breast cancer, heart disease, and stroke.

Those results led millions of women and their doctors to abandon HRT. Only later did researchers realize that the study’s design – focused largely on older, postmenopausal women – had skewed the results, leaving a generation of women in perimenopause without clear guidance or care.

The fallout from that moment still shapes the way that many women experience midlife care today – creating the gap that the Moslows are helping Tufts to fill.

Building from their catalyzing gift, with additional philanthropic support, the initiative aims to establish an academic model for compassionate, integrated menopause care, one connecting specialists in nutrition, cardiology, endocrinology, mental health, and sexual health.

Through Tufts Medicine’s network and telehealth programs, the hope is to share these benefits with women across Massachusetts.

In addition, the initiative will serve as a training ground for medical residents, dieticians, and other students across health sciences disciplines to learn evidence- and team-based approaches to care.

Education will be central to this work.

Tufts plans to embed menopause and women’s health content across curricula in its medical, physician assistant, physical therapy, and nutrition programs. Continuing and executive education offerings will extend this learning to practicing clinicians nationwide, amplifying Tufts’ role as a hub for best-practice training.

Tufts undergraduates also will have learning opportunities related to the program.

Research, too, will anchor the initiative’s efforts.

Across disciplines, Tufts scientists focus on longevity and healthy living. In the context of the Women’s Health and Menopause Initiative, they will explore vascular aging, neurobehavioral health, diet, and chronic disease.

They also seek to build a national menopause outcomes registry that links patient data, clinical care, and research, creating an unprecedented foundation for data-driven insights that will shape the future of women’s health.