Bold claim: 2025 was a watershed year for biomedical breakthroughs, delivering innovations that were once the stuff of science fiction and are now inching toward real-world impact. But here’s where it gets controversial: as rapid progress accelerates, debates about safety, ethics, and access intensify, reminding us that every leap comes with critical questions. This rewrite preserves the original meaning and key details while offering clearer, beginner-friendly explanations and fresh phrasing.
A year of firsts: BioXconomy Modalities’ standout innovations of 2025
2025 brought a stream of remarkable advances in biomedical science. From bispecific antibodies that target stage III melanoma to mRNA and antibody therapies tackling HIV, diseases once deemed hard to treat are facing new challenges to progress. The year showcased a broad spectrum of breakthroughs across therapies and technologies, underscoring a shift in what’s possible in medicine.
Below are reflections from Drug Modalities Editor Søren Hough (SH) and reporters Nnenna Ohaka (NO) and Millie Hoe (MH) on their favorite news stories of the year. (If you’re curious about prior years, you can read our 2024 list here.)
In a landmark achievement earlier in the year, researchers at the Children’s Hospital of Philadelphia and the University of Pennsylvania performed humanity’s first in vivo CRISPR therapy. They used a personalized mRNA-based approach to treat KJ, an infant with severe carbamoyl phosphate synthetase 1 deficiency (CPS1D). While this achievement represents a major breakthrough, it also sparked ongoing debates about genome editing’s therapeutic potential versus concerns about rushing into novel interventions, including broader questions about the normalization of “designer babies.” The promise for patients with otherwise debilitating conditions is undeniable, though the ethical discussions are far from settled. NO
For a deeper look, read about the n-of-1 study and the manufacturers’ perspectives on the therapy’s expedited development.
Batten’s disease, a rare genetic disorder that steals sight in early life, showed encouraging preclinical results in pig models. An antisense oligonucleotide (ASO) injected into the eye appeared to prevent retinal degeneration by preserving the cells responsible for light and color perception. While human trials remain distant, the finding that retinal activity persisted up to a year post-dosing offers a meaningful early signal. MH
Learn more about how this ASO could delay blindness in children with ultra-rare genetic conditions.
Science thrives on international collaboration, a principle clearly demonstrated in the development of a gene therapy for ADA-SCID, a rare immune disorder. Teams from University College London and the University of California, Los Angeles, joined forces to advance a therapy that entered clinical trials more than a decade ago. The collaboration has now shown safety and immune function restoration in 95% of treated patients. The next hurdle is ensuring access for those who need it, a challenge that sits at the intersection of science and the business models of pharma. SH
Read more about how transatlantic collaboration propelled this milestone.
A troubling statistic from Mayo Clinic data highlights disparities: Black women in the US face a 41% higher mortality rate from aggressive breast cancers despite a lower overall incidence, and they experience more delays in starting adjuvant chemotherapy. In this context, a triple-engineered antibody developed at King’s College London offers potential new options. In mouse models, this antibody attacked tumors effectively and slowed the growth of drug-resistant breast cancer, hinting at a therapy that could ultimately help address healthcare inequalities if it advances through development. NO
Explore how glycoengineering and Fc mutations enabled this promising approach.
A universal flu vaccine has long been a goal, and a collaborative effort between the Jackson Laboratory and Scripps Institute is pursuing it by targeting M2e, a viral component conserved across nearly all influenza A strains. If successful, a single vaccine could offer broad protection against many flu variants, a breakthrough as vaccination rates fluctuate and influenza deaths rise. MH
Discover how researchers are outsmarting the virus and reducing the chance of viral escape.
The field of fetal medicine continues to push boundaries, raising exciting yet contentious questions. In the realm of spinal muscular atrophy (SMA), a preclinical Science Translational Medicine study demonstrated that an ASO could treat SMA in utero in mice, with nusinersen (Spinraza) shown as a comparator. The researchers note that nusinersen may not be the ideal agent for in utero use and suggest that a more potent ASO could improve outcomes. This work highlights both the potential to intervene earlier in disease and the ethical considerations of treating unborn babies. SH
Read more about Sumner and her team’s prenatal SMA approach.
Ultra-rare diseases—though individually uncommon—affect millions collectively and often struggle to attract development investment. This year, eladocagene exuparvovec (Kebilidi) for aromatic L-amino acid decarboxylase (AADC) deficiency stood out as a major milestone: the first direct-to-brain AAV gene therapy approved in the US. In September 2025, the FDA issued Rare Disease Evidence Principles aimed at accelerating and clarifying reviews for therapies addressing rare diseases with small patient populations. If these guidelines hold, Kebilidi could reach some of the roughly 120 patients worldwide who could benefit. This signals a broader shift toward valuing ultra-rare diseases alongside more common conditions. NO
For more on Kebilidi, see the full coverage of the brain-directed gene therapy approval.
GLP-1s have grabbed headlines for obesity treatment, but recent research reveals cardiovascular benefits beyond weight loss. A meta-analysis from Harvard Medical School and the German Heart Centre, published in JAMA and drawing on data from over 58,000 patients, found that GLP-1 therapies reduced heart failure hospitalization and death risk—semaglutide by 42% and tirzepatide by 58%—independently of weight loss. This reframes how we think about these drugs’ benefits. MH
Learn about how GLP-1s may benefit heart health beyond their effects on body weight.
Sometimes headlines oversell a study, and a closer look reveals a different story. A viral PNAS Nexus paper prompted discussions about CRISPR and Down syndrome, but the lead author clarified that the work targets comorbidities rather than Down syndrome itself. The discussion offers a valuable window into the ethical landscape of genome editing and the ways research at the field’s edge can influence future directions. SH
Read the full article for nuanced context on this line of inquiry.
Among the year’s standout interests: tardigrades, the microscopic “water bears” famed for surviving extreme conditions, even in space. Scientists explored using a tardigrade protein called Dsup to shield normal tissue from radiation damage during cancer therapy. Early results indicate that delivering Dsup via mRNA-LNP could protect healthy cells while keeping tumors vulnerable to radiation, potentially reducing radiotherapy side effects. SH
Explore how this resilient organism’s biology might translate into patient benefits.
To our readers, thank you for supporting two years of BioXconomy coverage of emerging drug modalities. We’ve been encouraged by the warmth of your social media responses and in-person interactions, especially when BioXconomy joined forces with Tides Global in August 2025 to expand our modalities coverage. Here’s to a transformative 2026, with faster access to lifesaving therapies for patients in need.
Happy holidays and best wishes for the new year.
About the Authors
Nnenna Ohaka — Partnering and Investment Reporter at BioXconomy. With a biomedical sciences degree from the University of Warwick, she covers partnering and investment in drug modalities.
Millie Hoe — Life sciences Reporter for BioProcess Insider and BioXconomy, focusing on biomanufacturing, modalities, and partnering. She recently completed an MSc in biomedical sciences.
Søren Hough — Drug Modalities Editor at BioXconomy (formerly Tides Global). He earned a PhD in Biochemistry at the University of Cambridge, with research on DNA damage and repair, and he’s especially interested in the therapeutic potential and ethical dimensions of genome editing tools like CRISPR.
