A patient-specific CRISPR gene-editing therapy
A team at the Children鈥檚 Hospital of Philadelphia and Penn Medicine designed, tested, and delivered a for a rare metabolic disease, demonstrating that personalized genetic medicine can move from diagnosis to life-saving treatment at the speed of a crisis.
An FDA-approved therapy targeting the mitochondria
Stealth BioTherapeutics鈥� elamipretide received accelerated FDA approval as a an organelle long considered inaccessible to therapeutic intervention. Because mitochondria regulate core cellular functions, from energy production to metabolism and cell survival, this breakthrough suggests that one of biology鈥檚 most fundamental control centers may now be within therapeutic reach.
Positive data from a disease-modifying therapy for narcolepsy
In late-stage clinical trials, Takeda鈥檚 oveporexton , addressing the biological root cause of narcolepsy. Intervening at this control point signals the potential to shift away from symptom management for those living with this chronic neurological condition.
The first FDA-approved medicine for bronchiectasis
For decades, people living with bronchiectasis had no approved medicines to slow the progression of their disease. Insmed changed that with the approval of brensocatib, the , marking a shift from supportive care to disease modification.
A gene-based alternative replaces the scalpel
For patients with recurrent respiratory papillomatosis, treatment has meant frequent surgeries to remove benign tumors caused by the infection. Precigen鈥檚 Papzimeos suggests that these that retrains the immune system, changing what it means to live with this disease.
Disease-targeted therapies for IgA nephropathy
and advanced the first therapies designed to intervene in the specific immune and signaling pathways that drive IgA nephropathy, a leading cause of progressive kidney failure. By demonstrating that distinct biological mechanisms in IgA nephropathy can be selectively targeted, these advances reframe a nonspecific disease as one with identifiable, actionable drivers.
Human proof-of-concept for sustained impact of individualized cancer immunotherapy
, developed by Moderna in partnership with Merck, show that individualized neoantigen therapies (INT), when combined with checkpoint inhibition, can substantially reduce cancer recurrence in melanoma and lung cancer.
Extended local delivery of cancer medicine into the bladder
Johnson & Johnson鈥檚 Inlexzo received FDA approval as a therapy to , offering patients a potential surgery-free option. The therapy traces its origins to Taris Biomedical, which was developed in partnership with 蘑菇视频APP to rethink how drugs could be delivered to difficult-to-treat organs.
New ways to treat multiple myeloma
Cancer treatment is moving toward deeper biological control. Regeneron鈥檚 Lynozyfic, an , redirects a patient鈥檚 own immune cells to attack multiple myeloma without the delays and complexity of personalized cell therapies. Bristol Myers Squibb鈥檚 investigational medicine iberdomide in multiple myeloma, signaling the clinical maturation of targeted protein degradation as a way to eliminate disease-driving proteins rather than inhibit them.
Data supporting direct inhibition of RAS-driven cancers
For decades, oncogenic RAS mutations have driven some of the deadliest cancers while remaining beyond the reach of medicine. Revolution Medicines has that directly inhibit disease-driving RAS signaling, opening new treatment possibilities for cancers such as pancreatic and lung cancer.
Clinical proof in asthma for an AI-designed antibody
Flagship-founded Generate:Biomedicines designed and optimized a therapeutically viable antibody using AI algorithms, signaling a new mode of biologics invention. In severe asthma, , potentially allowing patients to manage a chronic condition with as few as two doses per year.
A new class of non-opioid painkiller
Vertex Pharmaceuticals introduced the first new class of pain medicine in more than two decades with Journavx (VX-548), . By offering effective pain relief without acting on opioid receptors, the drug demonstrates a viable path toward reducing reliance on drugs that can lead to addiction.
Transformative HIV prevention
Gilead Sciences鈥� lenacapavir can , addressing one of the most persistent barriers in global HIV prevention: sustained adherence. By pairing powerful antiviral biology with durability, the medicine shows how prevention strategies can be designed for a global scale.
Clinical proof for an oral PCSK9 inhibitor
For patients who need substantial LDL cholesterol reduction, the most effective therapies have required injections, limiting how widely they can be used in routine care. In Phase 3 trials, Merck鈥檚 investigational oral PCSK9 inhibitor enlicitide
Gene-edited pig kidneys enter human clinical trials
marks a turning point for xenotransplantation, moving the field from proof-of-concept science into clinical reality. By showing that organs engineered across species lines can be tested as viable transplants, this advance opened a path toward an abundant, reliable organ supply.
The broad impact of GLP-1 medicines
Decades of metabolic research culminated in GLP-1鈥揵ased medicines from Novo Nordisk and Eli Lilly that treat obesity through underlying biology. As these therapies reach population scale, are extending beyond weight loss, including improvements in cardiovascular, metabolic, and kidney disease.
Two new antibiotics overcome resistance pathways
New oral antibiotics from and demonstrate that novel mechanisms of action can successfully treat infections where existing therapies have steadily lost effectiveness. Together, they show that antibiotic resistance can be overcome with innovative approaches.
