In September 2025, Mighty marked ... treatment for Barth syndrome and the first FDA-approved therapy to directly target mitochondria. Today, Mighty's development portfolio encompasses rare and age-related diseases. Mighty continues to develop elamipretide in Barth syndrome and POLG-related mitochondrial disease. Mighty's Phase 3 ReNEW clinical trial of elamipretide ... In September 2025, Mighty marked a historic milestone with the U.S. Food and Drug Administration (FDA) approval of its first commercial therapy, establishing both the first FDA-approved treatment for Barth syndrome and the first FDA-approved therapy to directly target mitochondria. Today, Mighty's development portfolio encompasses rare and age-related diseases. Mighty continues to develop elamipretide in Barth syndrome and POLG-related mitochondrial disease. Mighty's Phase 3 ReNEW clinical trial of elamipretide in dry age-related macular degeneration is fully enrolled, with data expected late 2027, and Mighty is progressing its next-generation clinical candidate, bevemipretide, into a Phase 2/3 clinical trial in dry age-related macular degeneration by year-end. Trailblazing approach to leverage learnings from rare POLG disorders to inform development for Parkinson's and other age-related neurological diseases · NEEDHAM, Mass., Sept. 15, 2026 /PRNewswire/ -- Mighty Therapeutics (the "Company" or "Mighty"), a commercial-stage biotechnology company pioneering a new class of medicines that directly target mitochondrial dysfunction in rare and age-related diseases, is celebrating World Mitochondrial Disease Awareness Week by announcing today its initiation of a research collaboration with The POLG Foundation; Dr. The company was awarded a research grant in 2023 from the Michael J. Fox Foundation to evaluate bevemipretide, a next-generation mitochondria-targeted investigational therapeutic, in Parkinson's disease, for which it continues to evaluate dosing to inform potential clinical development. About The POLG Foundation The POLG Foundation is a 501(c)(3) non-profit research advocacy organization established in 2022 by a POLG family. The POLG Foundation raises awareness/resources to fuel research into POLG-related mitochondrial diseases. With the goal of developing effective treatments and a cure, The POLG Foundation connects researchers, clinicians, patients and families, key patient organizations and biopharma. "Current preclinical models are unable to recapitulate clinical aspects of Parkinson's disease or POLG disorders, presenting a significant barrier for new therapeutic development in either indication. This study brings together preclinical models of these two related diseases into a single model. If successful, the model could provide a new way to test for therapeutic efficacy, potentially paving the way for new mechanism-based treatments."

Drugs for aging-related diseases may modulate aging itself, but standard clinical trial designs cannot detect such effects. Aging clocks could close this gap, but epigenetic models often yield inconsistent, hard-to-interpret results. In contrast, proteomic clocks, by tracking the immediate ... Drugs for aging-related diseases may modulate aging itself, but standard clinical trial designs cannot detect such effects. Aging clocks could close this gap, but epigenetic models often yield inconsistent, hard-to-interpret results. In contrast, proteomic clocks, by tracking the immediate effectors of biological change, may excel in providing aging biomarkers or mechanistic insight. Nature Biotechnology - Six proteomic clocks are applied in a clinical trial to assess anti-aging effects. This work supports the goal of dual-purpose clinical trial designs that integrate aging endpoints into studies for specific disease indications. ... Discover the latest articles and news in related subjects. ... Decades of progress in identifying mechanisms of aging and therapeutic targets to modulate them have revealed a tremendous overlap with central regulators of disease. Both these precedents show how a biology-linked risk state can become a tractable therapeutic setting. We therefore propose a stepwise framework: first, collect aging and senescence biomarkers prospectively as exploratory endpoints in disease trials and explore the different dose and administration regimens; second, replicate effects in non-IPF age-related or risk-enriched populations where IPF disease modification is not the primary driver and third, pursue biomarker qualification or composite clinical endpoints to establish evidence of geroprotection.

🧬 Samsung Biologics launches all-cash tender offer to acquire PolyPeptide for ~CHF 1.46B ($1.81B), expanding peptide CDMO capabilities, UK biotech financing hits five-year high with £2.11B ($2.84B) equity investment in Q2 2026, Scribe Therapeutics plans $96M IPO to advance lipid-lowering ... 🧬 Samsung Biologics launches all-cash tender offer to acquire PolyPeptide for ~CHF 1.46B ($1.81B), expanding peptide CDMO capabilities, UK biotech financing hits five-year high with £2.11B ($2.84B) equity investment in Q2 2026, Scribe Therapeutics plans $96M IPO to advance lipid-lowering CRISPR genetic medicines Latigo Biotherapeutics plans IPO to fund non-opioid Nav1.8 inhibitor pain pipeline Pain management, small molecule, non-opioid, clinical-stage, Nav1.8 inhibitor - Read more · NuvOx Therapeutics files for $20M IPO, developing oxygen therapeutics targeting hypoxia-related diseases Clinical-stage, oxygen therapeutics, neurology, oncology, rare disease - Read more British Business Bank commits €25M ($28.6M) to EQT Life Sciences' EQT HE3 medtech and digital health fund Life sciences, medtech, digital health, venture capital - Read more · BlossomHill Therapeutics files for IPO, advancing small molecule oncology pipeline including EGFR-targeting BH-30643 Oncology, small molecule, clinical-stage, non-small cell lung cancer, leukemia - Read more · UK biotech financing hits five-year high with £2.11B ($2.84B) equity investment in Q2 2026 Biotech sector, financial, investment, venture capital - Read more Biotronik Neuro acquires Soin Neuroscience spinal cord stimulation patent portfolio for next-generation chronic pain program Acquisition, neurological, neuromodulation, chronic pain, medical devices - Read more · Kamada signs three-year $50M plasma supply agreement with unnamed biopharmaceutical company Manufacturing agreement, plasma-derived therapies, rare disease, commercialization, milestone payments - Read more

Spero Therapeutics is putting over $1 billion on the line in exchange for rights to develop SP001, an anti-CD40L antibody, for IgG4-related disease. A Phase 2 study is planned for the second quarter of 2027.

Longevity is a long-standing buzzword in life sciences, but it now has staying power. The smart trajectory is to stop chasing aging as an abstract target and concentrate on specific mechanisms that can clearly advance specific, age-related diseases, according to two investors in a discussion with BioSpace.