A patient-specific CRISPR gene-editing therapy
A team at the Children’s Hospital of Philadelphia and Penn Medicine designed, tested, and delivered a gene-editing therapy tailored to a single infant’s mutation 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’ elamipretide received accelerated FDA approval as a medicine that can directly target mitochondria, 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’s 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’s oveporexton restored signaling in the brain’s central regulator of wakefulness and sleep stability, 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 first treatment to directly intervene in the inflammatory processes that drive chronic lung damage, 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’s Papzimeos suggests that these tumors can instead be controlled through a targeted genetic signal that retrains the immune system, changing what it means to live with this disease.
Disease-targeted therapies for IgA nephropathy
Novartis and Otsuka 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
Late-stage clinical trials of mRNA-4157, 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’s Inlexzo received FDA approval as a therapy to deliver cancer medicine directly into the bladder over an extended period, offering patients a potential surgery-free option. The therapy traces its origins to Taris Biomedical, which was developed in partnership with Flagship Pioneering 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’s Lynozyfic, an off-the-shelf bispecific antibody, redirects a patient’s own immune cells to attack multiple myeloma without the delays and complexity of personalized cell therapies. Bristol Myers Squibb’s investigational medicine iberdomide demonstrated positive Phase 3 results 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 advanced therapies now in late-stage clinical trials 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, GB-0895 shows how this approach can translate into long-acting disease control, 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), an oral therapy that prevents pain signals from reaching the brain. 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’ lenacapavir can prevent HIV infection with just two injections per year, 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’s investigational oral PCSK9 inhibitor enlicitide indicates that this level of cholesterol lowering can be achieved with a once-daily pill.
Gene-edited pig kidneys enter human clinical trials
FDA authorization of human clinical trials for gene-edited pig kidneys 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–based medicines from Novo Nordisk and Eli Lilly that treat obesity through underlying biology. As these therapies reach population scale, studies are revealing benefits extending beyond weight loss, including improvements in cardiovascular, metabolic, and kidney disease.
Two new antibiotics overcome resistance pathways
New oral antibiotics from GSK and Innoviva 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.
