Library
Medical & science breakthroughs
Twice-yearly HIV shot approved: lenacapavir (Yeztugo)
On June 18, 2025, the U.S. FDA approved Gilead Sciences' Yeztugo (lenacapavir), an injectable HIV 1 capsid inhibitor, as pre exposure prophylaxis (PrEP) — the first and only twice yearly PrEP option in the United States, for adults and adolescents weighing at least 35 kg. In the Phase 3 PURPOSE 1 trial, zero HIV infections occurred among 2,134 participants (a 100% reduction), and in PURPOSE 2, 2 infections occurred among 2,179 participants — more than 99.9% of all trial participants remained…
Malaria vaccines reach 20+ African countries — and deaths are falling
By 2026, more than 20 African countries had integrated malaria vaccines (RTS,S/AS01 and R21/Matrix M) into routine immunization programs, with over 24 million doses delivered (malaria vaccine tracker, updated July 2026). Uganda launched Africa's largest campaign in March 2025 — 2.278 million R21 doses across 105 districts targeting 1.1 million children under 2. Zambia became the 24th country to introduce the vaccine (October 2025), targeting 500,000+ children. On December 15, 2025, MSF…
A young man functionally cured of sickle cell disease — and the cure is reaching children
Daniel Cressy, 23, of Metairie, Louisiana, became the first person in Louisiana and the Gulf South to be functionally cured of sickle cell disease. His stem cells were collected in late 2025 (collection began July 30, 2025), shipped to Scotland for CRISPR editing with Casgevy (exagamglogene autotemcel), and returned in March 2026; after chemotherapy to clear his diseased cells, the edited cells were infused on March 18, 2026, at Manning Family Children's Hospital in New Orleans. Discharged in…
Lab-grown islet cells free type 1 diabetes patients from insulin
Vertex Pharmaceuticals' zimislecel (formerly VX 880) — a one time infusion of stem cell derived, fully differentiated islet cells into the hepatic portal vein — delivered striking Phase 1/2 results in the FORWARD trial: all 12 patients with type 1 diabetes and impaired hypoglycemic awareness showed restored insulin secretion (C peptide); all 12 eliminated severe hypoglycemic events; all met glycemic targets (A1C below 7%, time in range above 70%); exogenous insulin use fell by an average of…
China's NEO: world's first commercial brain implant for paralyzed patients
On March 13, 2026, China's National Medical Products Administration (NMPA) approved the registration application for Neuracle Technology's NEO — an implantable brain computer interface (BCI) for hand motor augmentation — marking the world's first market launch of an invasive BCI medical device (reported by Xinhua via People's Daily, March 13, 2026). The coin sized wireless device is implanted above the dura mater (not penetrating brain tissue), decoding brain signals in real time to drive a…
Eight healthy babies born with "three-person" mitochondrial donation IVF
On July 16, 2025, Newcastle University announced that eight babies — four girls and four boys, including one set of identical twins — had been born to seven women at high risk of transmitting serious mitochondrial DNA disease, using the licensed IVF technique of mitochondrial donation (pronuclear transfer). The findings were published in two papers in the New England Journal of Medicine. All eight babies were healthy at birth, are meeting their developmental milestones, and show no signs of…
US cancer survival hits 70% for the first time — 4.8 million deaths averted since 1991
On January 13, 2026, the American Cancer Society released Cancer Statistics, 2026 (published in CA: A Cancer Journal for Clinicians): for the first time, the five year relative survival rate for all cancers combined reached 70% for people diagnosed during 2015–2021 in the United States — up from 50% in the mid 1970s and 63% in the mid 1990s. Survival gains since the mid 1990s are largest for historically fatal cancers: multiple myeloma (32% to 62%), liver cancer (7% to 22%), lung cancer (15% to…
Baby KJ: the first person treated with CRISPR built just for him — walking and thriving at one year
KJ (Kyle Muldoon Jr.), born August 1, 2024, in Philadelphia, was diagnosed with severe neonatal onset CPS1 deficiency — a urea cycle disorder so rare (about one in a million) it is fatal for roughly half of affected infants; the only long term treatment was a liver transplant. Physicians at Children's Hospital of Philadelphia and Penn Medicine (Drs. Rebecca Ahrens Nicklas and Kiran Musunuru) designed a personalized CRISPR base editor for KJ's exact mutation in about six months; after FDA…
Kisunla: the first drug shown to slow Alzheimer's decline gets FDA approval
On July 2, 2024, the U.S. FDA approved donanemab (marketed as Kisunla), an amyloid targeting infusion, for adults with early symptomatic Alzheimer's disease — the second anti amyloid drug to reach approval after lecanemab. In the Phase 3 TRAILBLAZER ALZ 2 trial, donanemab slowed clinical decline by about 22% overall and by up to 35% in participants with less advanced disease, reducing the risk of disease progression (Clinical Dementia Rating scale) by roughly 37%. The drug carries a boxed…
Zurzuvae: the first oral medicine for postpartum depression
On August 4, 2023, the U.S. FDA approved zuranolone (marketed as Zurzuvae), the first ever oral medication specifically for postpartum depression (PPD). It is taken as a once daily, 14 day course. In clinical trials, symptom improvement was seen as early as day 3 and was maintained after treatment ended — a stark contrast to standard antidepressants, which typically take weeks to work and must be taken indefinitely. The FDA did not approve it for major depressive disorder more broadly; that…
Cobenfy: a genuinely new kind of schizophrenia drug — the first in decades
On September 26, 2024, the U.S. FDA approved Cobenfy (xanomeline trospium), the first approved schizophrenia medicine that targets muscarinic receptors instead of the dopamine receptors that every previous antipsychotic has worked through. It is the first drug with a novel mechanism of action for schizophrenia approved in several decades. In the Phase 3 EMERGENT trials, Cobenfy produced statistically significant reductions in schizophrenia symptoms versus placebo, with a side effect profile…
Gene therapy gives deaf children their hearing back
In a study published June 5, 2024, in Nature Medicine, researchers reported that bilateral gene therapy for OTOF related deafness restored hearing and improved speech perception and sound localization in all five children in the initial bilateral trial cohort — with no serious adverse events. The therapy delivers a working copy of the otoferlin (OTOF) gene, whose mutations cause a form of congenital deafness, directly to the inner ear. A separate later study in ten participants also reported…
Roctavian: the first U.S. gene therapy for severe hemophilia A
On June 29, 2023, the U.S. FDA approved BioMarin's Roctavian (valoctocogene roxaparvovec), the first and only gene therapy for adults with severe hemophilia A in the United States. A single one time infusion delivers a functional copy of the factor VIII gene, enabling patients' own bodies to produce the missing clotting factor. In the FDA analysis of the pivotal trial, treated patients saw their mean annualized bleeding rate fall by about 52%.
Cystic fibrosis predicted survival reaches 65 years — up from 46
According to 2024 registry data reported in early 2026, the predicted survival for people with cystic fibrosis rose to 65 years for the 2020–2024 period, up from 46 years for 2015–2019. The jump reflects the cumulative effect of highly effective CFTR modulator therapies (such as Trikafta), earlier newborn screening, and better multidisciplinary care — and it is measured as a real population health outcome, not a trial endpoint. For a disease that was once routinely fatal in childhood, the…
A single shot is keeping babies out of the hospital with RSV
U.S. surveillance data for the 2024–2025 RSV season showed that infant hospitalization rates were 28% and 43% lower in two national surveillance networks following the rollout of nirsevimab (Beyfortus), the long acting monoclonal antibody given as a single injection to protect infants through their first RSV season. A subsequent effectiveness study estimated nirsevimab was about 81% effective against RSV hospitalization, with similarly promising estimates against severe outcomes. The result is…
The first new tuberculosis vaccine in a century shows ~50% protection
The M72/AS01E tuberculosis vaccine candidate demonstrated approximately 50% protection against TB disease over three years of follow up in its Phase 2b trial — the first meaningful efficacy signal for a new TB vaccine in a century (the only licensed TB vaccine, BCG, dates to 1921 and protects poorly against adult pulmonary TB). A global Phase 3 trial of M72/AS01E began in March 2024 and reached full enrollment of 20,000 participants in April 2025. The story here is the completed Phase 2b…
A personalized mRNA vaccine cuts melanoma recurrence by 44%
In Phase 2b results, the personalized mRNA cancer vaccine mRNA 4157 (V940), given in combination with pembrolizumab (Keytruda), reduced the risk of melanoma recurrence or death by 44% compared with pembrolizumab alone in patients with high risk resected melanoma. At 18 months, recurrence free survival was 78.6% with the combination versus 62.2% with pembrolizumab alone. The vaccine is custom built for each patient from the mutations in their own tumor — up to 34 neoantigens per dose. The…
Pancreatic cancer patients still cancer-free years after a personalized vaccine
In a small Phase 1 trial of a personalized mRNA vaccine for pancreatic cancer — one of the deadliest cancers, with most patients relapsing after surgery — six of the eight patients whose immune systems responded to the vaccine remained cancer free at a median follow up of 3.2 years. Later reporting put it even more strikingly: seven of the eight immune responders were still alive four to six years after surgery. The vaccine, tailored to each patient's tumor mutations, was given after surgery…
Journavx: the first new kind of painkiller in 20+ years — and it's not an opioid
On January 30, 2025, the U.S. FDA approved Journavx (suzetrigine), the first new class of medicine for acute pain in more than two decades — and the first non opioid in that category. It works by blocking NaV1.8, a sodium channel involved in pain signaling, rather than acting on opioid receptors. In trials it beat placebo for acute pain relief; it did not clearly outperform opioids overall, and it should not be framed as "better than opioids." Its value is as an approved, effective non opioid…
Veozah: the first nonhormonal treatment for menopause hot flashes
On May 12, 2023, the U.S. FDA approved Veozah (fezolinetant), the first NK3 receptor antagonist — and the first nonhormonal treatment — for moderate to severe vasomotor symptoms (hot flashes and night sweats) of menopause. In Phase 3 trials, it significantly reduced both the frequency and severity of hot flashes versus placebo. For women who cannot or prefer not to take hormone therapy, it created the first targeted nonhormonal option. (The drug carries liver safety labeling; patients need…
CRISPR editing inside the body slashes the protein behind ATTR amyloidosis
NTLA 2001, an in vivo CRISPR based therapy for transthyretin (ATTR) amyloidosis, produced striking early results: a single infusion drove roughly 87–93% mean reductions in the disease causing TTR protein in higher dose cohorts. In a 2024 report on the cardiomyopathy population, 92% of treated patients maintained or improved their NYHA heart failure class, with average one year TTR reduction of about 90%. ATTR amyloidosis — in which misfolded TTR protein builds up in the heart and nerves — is…
One CRISPR dose nearly eliminated swelling attacks in hereditary angioedema
In a Phase 2 trial of 27 adults with hereditary angioedema (HAE) — a genetic disease causing unpredictable, potentially life threatening swelling attacks — a single 50 mg dose of the in vivo CRISPR therapy NTLA 2002 reduced monthly attack rates by 77–81%, and 8 of 11 patients in the key cohort were completely attack free during the main observation window. No treatment attributed serious adverse events were reported. The therapy works by editing the KLKB1 gene in the liver to durably lower…
Every rectal cancer patient in the trial saw their tumor vanish on immunotherapy
In an updated analysis presented at ASCO 2024 from Memorial Sloan Kettering's Phase 2 study, the immunotherapy dostarlimab (Jemperli) produced a 100% clinical complete response rate in 42 patients with mismatch repair–deficient (dMMR) locally advanced rectal cancer who completed treatment — defined as no evidence of tumor on MRI, endoscopy, and digital rectal exam, with no surgery, radiation, or chemotherapy needed. In the first 24 patients evaluated, the complete response was sustained with a…
Amtagvi: the first cell therapy for a solid tumor — and responses lasting years
On February 16, 2024, the U.S. FDA granted accelerated approval to Amtagvi (lifileucel), the first tumor derived T cell (TIL) therapy approved for any solid tumor — for adults with unresectable or metastatic melanoma previously treated with PD 1 blockade. In the pivotal C 144 01 trial, the objective response rate was 31.5% in the primary cohort, including complete responses, in patients who had run out of options. The final five year analysis (Journal of Clinical Oncology, 2025) showed…
Lorlatinib: 60% of ALK lung cancer patients progression-free at five years
The five year analysis of the Phase 3 CROWN trial, presented at ASCO 2024 and published in the Journal of Clinical Oncology, reported that lorlatinib (Lorbrena) kept 60% of patients with previously untreated ALK positive advanced non small cell lung cancer alive without disease progression at five years — versus just 8% with the prior standard crizotinib. The hazard ratio of 0.19 translates to an 81% reduction in the risk of progression or death, and median progression free survival had not…
Winrevair: a first-of-its-kind drug transforms pulmonary arterial hypertension
On March 26, 2024, the U.S. FDA approved Winrevair (sotatercept), the first activin signaling inhibitor therapy for pulmonary arterial hypertension — a first of its kind drug for a rare, progressive, and often fatal lung disease. In the pivotal STELLAR trial, patients on Winrevair added to background therapy walked 41 meters farther in the six minute walk test and had an 84% lower risk of death or clinical worsening compared with background therapy alone. The label was expanded on October 27,…
Lenmeldy: gene therapy stops a fatal childhood brain disease
On March 18, 2024, the U.S. FDA approved Lenmeldy (atidarsagene autotemcel), the first gene therapy for children with metachromatic leukodystrophy (MLD) — a rare, inherited, rapidly fatal disease that destroys the brain's white matter in early childhood. The results in presymptomatic children were extraordinary: in the pivotal studies of 39 children, all treated presymptomatic late infantile patients were alive at age six compared with 58% of untreated historical controls; 71% were walking…
Vyjuvek: a gene-therapy gel heals wounds in "butterfly" children
In May 2023, the U.S. FDA approved Vyjuvek (beremagene geperpavec), the first redosable gene therapy — a topical gel for dystrophic epidermolysis bullosa (DEB), the "butterfly skin" disease in which the slightest friction tears the skin into chronic, painful wounds. In the Phase 3 GEM 3 trial, 65% of treated wounds were completely closed at six months versus 26% with placebo, and 68% versus 23% at three months — with no treatment related serious adverse events reported in the extension…
Beqvez: a one-time gene therapy for hemophilia B (with a commercial caveat)
On April 26, 2024, the U.S. FDA approved Beqvez (fidanacogene elaparvovec), a one time gene therapy for adults with hemophilia B. In the pivotal BENEGENE 2 study, the mean annualized bleeding rate fell to 2.5 after treatment versus 4.5 during the prophylaxis lead in; bleeds were eliminated entirely in 60% of patients versus 29% on standard factor prophylaxis, and the median annualized bleeding rate was zero. In February 2025, Pfizer announced it was ending global development and…
Tirzepatide cut progression from prediabetes to diabetes by 94%
In the three year (176 week) analysis of the SURMOUNT 1 trial, published in the New England Journal of Medicine in November 2024, tirzepatide (Zepbound/Mounjaro) reduced progression from prediabetes to type 2 diabetes by 94% (hazard ratio 0.06) in 1,032 adults with prediabetes and overweight or obesity. Only 1.2% of tirzepatide treated participants developed diabetes versus 12.6% on placebo — meaning nearly 99% remained diabetes free — alongside sustained weight loss of up to 22.9% at the…
Osimertinib after surgery: lung cancer survival hits 85–88%
The five year overall survival analysis of the Phase 3 ADAURA trial (presented at ASCO 2023, published in the New England Journal of Medicine) showed that adjuvant osimertinib (Tagrisso) — a daily pill taken after surgery for EGFR mutated non small cell lung cancer — raised five year overall survival to 85% versus 73% with placebo in stage II–IIIA disease (hazard ratio 0.49), and to 88% versus 78% across the full stage IB–IIIA population. It was the first targeted therapy ever to demonstrate an…
ARC-EX: the first non-invasive stimulation that restores hand function after spinal cord injury
On December 19, 2024, the U.S. FDA granted de novo classification and market authorization to ONWARD Medical's ARC EX System — the world's first non invasive spinal cord stimulation system for people with chronic spinal cord injury. The system delivers programmed electrical stimulation through the skin to the spinal cord via electrodes on the back of the neck, with no surgery required. The authorization followed the multisite Up LIFT pivotal trial, which showed the technology improves hand…
Chad eliminates sleeping sickness as a public health problem
On June 20, 2024, the World Health Organization announced that Chad had eliminated the gambiense form of human African trypanosomiasis (sleeping sickness) as a public health problem — the first neglected tropical disease to be eliminated in the country. Chad became the 51st country recognized by WHO for eliminating at least one neglected tropical disease, passing the halfway mark toward the global target of 100 countries. Sleeping sickness is a parasitic disease transmitted by tsetse flies that…
India eliminates trachoma as a public health problem
On October 8, 2024, the World Health Organization declared that India had eliminated trachoma as a public health problem, making it the third country in the South East Asia region to reach the milestone. Trachoma is a bacterial eye infection that was once a leading cause of blindness; India's National Trachoma Control Programme, running since 1963, drove blindness attributable to trachoma down from 5% in 1971 to under 1% today. The declaration reflects verified prevalence below WHO elimination…
Egypt becomes the first country to reach WHO's Gold Tier on the path to hepatitis C elimination
In October 2023, Egypt became the first country in the world to achieve the World Health Organization's Gold Tier status on the path to hepatitis C elimination. The program is staggering in scale: more than 60 million people screened and over 4.3 million cured, driving national prevalence down from around 10% to 0.38%. "Gold Tier" means on the path to elimination — not full elimination — and it is framed that way here. The achievement rests on mass screening, locally produced low cost direct…
Stem cells from a patient's own eye restore vision after corneal damage
In results published March 4, 2025, in Nature Communications, the CALEC procedure — transplanting stem cells grown from a patient's own healthy eye to repair a damaged cornea — succeeded in nearly all treated patients. In the trial, 14 patients were followed for 18 months: complete corneal restoration reached 50% at three months and 79% at 12 months; overall complete or partial success was 93% at 12 months and 92% at 18 months; and all 14 patients experienced at least some improvement in visual…
Semaglutide cuts kidney failure and death in people with diabetes and kidney disease
In the Phase 3b FLOW trial — 3,533 adults with type 2 diabetes and chronic kidney disease — weekly semaglutide 1.0 mg, reported in the New England Journal of Medicine in May 2024, reduced the composite of major kidney events, cardiovascular death, and kidney death by 24%, cut major adverse cardiovascular events by 18%, and lowered all cause mortality by 20% versus placebo. The trial was stopped early in October 2023 on the recommendation of its data monitoring committee after an interim…
Stem-cell-derived neurons show durable benefit in Parkinson's after three years
In 36 month results from the Phase 1 trial of bemdaneprocel — stem cell derived dopamine neurons transplanted into the brains of people with Parkinson's disease — the therapy showed no therapy or surgery related adverse events and durable motor improvements: high dose participants improved by a mean of 17.9 points on the off medication MDS UPDRS Part III motor scale, and low dose participants by 13.5 points, both clinically meaningful changes. Brain imaging confirmed the transplanted cells…
A New York woman's HIV stayed gone 18 months after a stem-cell transplant
In a paper published in Cell on March 16, 2023, researchers reported that a New York woman with HIV 1 — the "New York patient" — remained in remission with no viral rebound for at least 18 months after stopping antiretroviral therapy, following a CCR5 delta32/delta32 haplo cord stem cell transplant performed to treat her concurrent leukemia. No HIV 1 DNA or RNA and no replication competent virus were detectable. She was the first mixed race woman documented in HIV remission. The transplant was…
Guinea worm cases hit a historic low — 10 in all of 2025
On January 30, 2026, the Carter Center announced that only 10 human cases of Guinea worm disease were reported worldwide in all of 2025 — the lowest annual total ever recorded for a disease that afflicted an estimated 3.5 million people a year in the 1980s. The 2024 total had been 15 human cases (9 in Chad, 6 in South Sudan), itself a record low at the time. Animal infections — the main remaining reservoir — also fell, down 25% in the 2024 reporting period (from 887 to 664). Eradication, once a…