Happy New Year, nerds. While you were doom-scrolling through 2025, biotech was quietly making moves. We had a baby receive the world’s first personalized CRISPR therapy, an HIV prevention shot so effective it made clinical trialists do a double-take, and two innovations that sound simple but have eluded us for decades. Consider this our love letter to a year that delivered.
Table of Contents
🧬 The FDA cleared a custom gene therapy in one week. Yes, you read that right.
RESEARCH
In February 2025, a nearly seven-month-old baby named KJ Muldoon became the first human to receive a personalized CRISPR-based gene therapy, engineered specifically for his unique genetic mutation. Turns out, when your patient has a condition affecting 1 in 1.3 million newborns with a 50% infant mortality rate, bureaucracy can move fast.
KJ was born with severe CPS1 deficiency, an ultra-rare disorder where the liver can't process ammonia. Without intervention, the toxic buildup causes brain damage. His physicians at Children's Hospital of Philadelphia partnered with Kiran Musunuru at Penn and the Innovative Genomics Institute at Berkeley to design a bespoke adenine base editor, lovingly named "kayjayguran," that would correct one faulty DNA letter out of 3 billion.
The timeline was staggering. From diagnosis to FDA clearance: six months. The FDA reviewed the application in one week (apparently, they do emergency speeds when babies are dying). The treatment used lipid nanoparticles, the same delivery tech as COVID vaccines, which meant KJ could receive multiple doses without the immune system freaking out.
By June 2025, after 307 days in the hospital, KJ went home. He's hitting developmental milestones, fighting off colds without dangerous ammonia spikes, and has already been named to Nature's "10 People Who Shaped Science in 2025" list. He's not cured (his team was careful to clarify that), but he's thriving. The template they built could theoretically work for any of the thousands of rare genetic diseases lacking treatments. Personalized medicine went from buzzword to reality this year, and it started with one tiny patient.
💉 A twice-yearly HIV shot that worked so well, researchers had to stop the trial early
RESEARCH & NEWS
In June 2025, the FDA approved Yeztugo (lenacapavir) as the first twice-yearly injectable PrEP, and the efficacy data borders on absurd. The PURPOSE 1 trial in African women? Zero infections among 2,134 participants. Not low. Zero. The study was stopped early because it would have been unethical to keep giving people placebos.
Gilead's capsid inhibitor works differently than anything else in the HIV arsenal. It physically locks up the viral capsid protein at picomolar potency, blocking multiple stages of infection simultaneously. This is apparently what happens when you design a drug that works at concentrations so low they're barely measurable.
The PURPOSE 2 trial in men who have sex with men and transgender individuals showed 89% greater protection than daily Truvada, because of course it did. Daily pills have an adherence problem: roughly half of users quit within a year. Two injections annually? Much harder to forget.
The equity angle is where it gets interesting. US list price: $28,218 per year (this is America, after all). But through partnerships with the Gates Foundation and generic manufacturers, low-income countries will pay $40 per year starting in 2027. WHO prequalified the drug in record time, just 36 days. With 1.3 million new HIV infections still occurring annually, this could fundamentally alter the epidemic's trajectory.
🔥 Non-hormonal menopause relief finally exists, and breast cancer survivors can actually use it
NEWS
In October 2025, the FDA approved Lynkuet (elinzanetant), Bayer's first-in-class dual NK1/NK3 receptor antagonist for hot flashes. Translation: it's a pill that tells your hypothalamus to stop launching surprise personal summers, and it does so without touching your estrogen levels.
This matters enormously for a population that's been quietly suffering. Up to 80% of breast cancers are estrogen receptor-positive, meaning hormone replacement therapy is contraindicated. Same for women with blood clots history or cardiovascular disease. These patients have been stuck choosing between misery and medical risk. Elinzanetant targets the KNDy neurons (the delightful brain cells that go haywire when estrogen drops) by blocking both NK-1 and NK-3 receptors.
The OASIS trial program delivered the goods: 73% reduction in hot flash frequency at 12 weeks versus 47% for placebo. Over 70% of patients achieved at least a 50% reduction in symptoms. The OASIS-4 trial specifically studied breast cancer patients on tamoxifen or aromatase inhibitors, confirming that it works for the patients who need it most.
There are caveats: it requires liver monitoring, can cause drowsiness, and won't help with vaginal dryness or bone density as HRT does. But for the millions of women who couldn't safely take hormones, this represents the first real pharmaceutical option that isn't "have you tried evening primrose oil?"
👃 After 35 years, someone finally figured out needle-free epinephrine
NEWS
Neffy's pediatric expansion in March 2025 deserves a spot on this list purely for the "why did this take so long" factor. ARS Pharmaceuticals created a nasal spray epinephrine that kids as young as four can receive, and the implications for the approximately 1-in-17 children with food allergies are significant.
The problem with EpiPens isn't the drug, it's the delivery. 56% of caregivers report fearing they'll hurt their child using one. Needle phobia affects up to 63% of children, and in anaphylaxis, delayed treatment means worse outcomes. Studies consistently show epinephrine is dramatically underused during allergic emergencies, with only about 20% of children receiving pre-hospital epinephrine when warranted.
Neffy sidesteps all of this. One spray. One nostril. Done. Human factors studies showed 100% successful administration by untrained users. The nasal spray uses an absorption enhancer called Intravail that temporarily opens gaps between cells, getting epinephrine into the bloodstream in under a minute. Real-world data from 545 patients showed 89% were effectively treated with a single dose, matching historical EpiPen performance.
It's also half the size of an iPhone and costs $199 for a two-pack versus $650+ for brand-name EpiPen. Shelf life? 24 months versus 12-18 for EpiPen. Temperature tolerance? Survives 122°F for months while EpiPens degrade outside room temperature. Schools can now stock something that babysitters, teachers, and panicking parents can actually use correctly. Sometimes the most revolutionary breakthroughs are the simplest ones.
🎆 That's a wrap on 2025
Four stories. Four genuine advances that will change how we treat disease, prevent infection, manage symptoms, and save lives in emergencies. Not bad for a single trip around the sun.
To our 2,000+ readers maintaining that absurd 70%+ open rate: thank you for caring about this stuff. We do it because you actually read it, forward it, and occasionally yell at us when we get the mechanism of action wrong. (We love that, genuinely.)
Here's to a 2026 with even weirder science.
Keep questioning everything, and happy new year,