When results for a new pancreatic cancer pill were presented at the American Society of Clinical Oncology’s annual meeting in May, the audience gave them a rare, 42s standing ovation. It was one of a string of medical advances this year, several built on gene editing and AI, that have mostly stayed in the specialist press.
An X user who goes by Mgoes is trying to change that. “This might be my last normal tweet before biotech goes vertical,” Mgoes posted on 27 October, listing 10 advances. The news, Mgoes wrote, was “sitting in press releases and almost nobody reads them”. Two days later, Elon Musk shared the post: “If you look closely enough, breakthroughs in everything are accelerating exponentially. An exponential always starts off looking small.”
The account is pseudonymous, lists no scientific credentials and describes its owner as an investor in SuperHuman Fund, a “human enhancement” venture fund that co-hosts the Accelerate Bio podcast and has backed at least one guest’s company.
Not every item on the list is a breakthrough, and only about half rely on gene editing or AI. But together they show where both technologies are starting to reach patients.
If you look closely enough, breakthroughs in everything are accelerating exponentially. An exponential always starts off looking small.
Elon Musk
Gene editing changes the DNA instructions inside cells. Crispr-Cas9, the “genetic scissors” that won Emmanuelle Charpentier and Jennifer Doudna the 2020 Nobel Prize in chemistry, became a medicine in late 2023, when UK and US regulators cleared Casgevy for sickle cell disease. Base editing, a newer version, works more like fixing a typo than cutting out a sentence.
Google DeepMind’s Demis Hassabis and John Jumper won half of the 2024 chemistry Nobel for AlphaFold, which predicts the shapes of virtually all 200 million known proteins, helping scientists work out what they do. AI now also picks drug targets and designs molecules to hit them.
What this year has delivered
- A pill that helps people with advanced pancreatic cancer live longer. Revolution Medicines’ daraxonrasib roughly doubled median survival in a trial of 500 patients whose cancer had spread despite earlier treatment: 13.2 months, against 6.7 months on chemotherapy. The US Food and Drug Administration (FDA) approved it as Rasonque in August. It blocks faulty RAS proteins, which the company says drive more than 90% of pancreatic cancers.
- A one-off gene-editing drip that prevents most attacks of a painful swelling disorder. In April, Intellia Therapeutics said a single infusion of its CRISPR treatment, lonvo-z, cut attacks of hereditary angioedema by 87% compared with a placebo. It edits a gene inside the body to cut production of a protein that triggers the swelling, and 62% of patients were attack-free and needed no other treatment for six months. It is not risk-free: US regulators paused trials of Intellia’s other in-body treatment for several months after a patient suffered serious liver damage and later died.
- A single treatment to lower cholesterol, possibly for life. VERVE-102, which uses base editing to switch off a liver gene called PCSK9, lowered “bad” cholesterol by 62% at the highest dose in an early trial of 35 people, researchers from Eli Lilly subsidiary Verve Therapeutics reported. Reductions lasted up to 18 months, though the longest follow-up so far is in patients on lower doses.
- The next generation of weight-loss jabs. In May, Lilly said its weekly injection retatrutide helped people on the top dose lose an average of 25% of their body weight over 80 weeks, counting everyone who started treatment, though 11.3% on that dose quit because of side effects. It is a conventional drug that mimics three appetite and blood-sugar hormones, where Ozempic mimics one.
- A lung drug found and designed with AI. In September, Insilico Medicine began final-stage (phase 3) testing in China of rentosertib for idiopathic pulmonary fibrosis, a disease that steadily scars the lungs. AI sifted biological data to flag a target, an enzyme called TNIK, and generative AI designed a drug to block it. “TNIK, the target driven by AI, had never previously been linked to fibrosis,” the trial’s lead investigator, Prof Zuojun Xu, said in Insilico’s announcement.
- A vaccine made for one patient’s cancer. Computer programs pick up to 34 mutated targets unique to each patient’s tumour, and a messenger RNA (mRNA) vaccine trains the immune system to hunt them. In October, Merck and Moderna said their vaccine, given with the immunotherapy drug Keytruda, cut the risk of melanoma returning, or of death, by 42% compared with Keytruda alone, in a trial of 1 137 patients. It cut the risk of the cancer spreading, or of death, by 38%, less than the 59% in an earlier, smaller trial.
- A pill to regrow hair. In October, Veradermics said its pill met all its goals in a second late-stage trial in men. It is minoxidil, the blood-pressure drug behind Rogaine, reworked as a slow-release tablet.
- Cancer treatment without the knife, for some. Also in October, the FDA granted early, conditional approval to GSK’s dostarlimab for a type of rectal cancer whose many genetic errors make it easier for the immune system to spot, once the drug takes the brakes off. Of 154 patients in the main trial, 71% were treated with dostarlimab alone, needed no surgery or other treatment and saw no progression of their cancer.
- A pig kidney as a bridge to transplant. Tim Andrews lived a record 271 days without dialysis on a pig kidney carrying 69 genetic edits, until it was removed in October 2025 as its function declined. In January he received a human kidney, and The Lancet has since published the case, which his hospital describes as the first pig-kidney bridge to a human transplant.
- Cells that share DNA. This is basic science, not medicine. A May study in Cell found chunks of DNA moving between cells through tiny tubes and carrying traits to the cells that received them. But this was lab work: the researchers mixed two cell types and introduced errors or damage into their DNA. Whether it happens in the body is unknown.
The pancreatic cancer, swelling, cholesterol and weight-loss results are published in the New England Journal of Medicine, and the rectal cancer results in Annals of Oncology. Only the hair-loss results rest on company data alone. Several trials are early, small or uncontrolled, and only two of the treatments have been approved.
A 2024 analysis by Boston Consulting Group researchers counted 75 AI-discovered molecules entering human trials between 2015 and 2023, a number growing by more than 60% a year. They passed their first safety trials 80-90% of the time, “substantially higher than historic industry averages”. But in the larger phase 2 trials that test whether a drug works, only four of 10 succeeded, “comparable to historic industry averages”.
No drug with both an AI-found target and an AI-designed molecule has been approved. Xu expects rentosertib to need three to four years from the start of phase 3 to approval, “under favourable conditions”.
Gene editing has moved faster, from its first approved therapy, Casgevy, to a successful final-stage trial of an in-body edit, lonvo-z, in under two and a half years. So far, Musk’s exponential shows up in the number of new treatments entering trials, not yet in the number reaching patients.
What it means for South Africa
We found no record that any of the 10 treatments is registered with the South African Health Products Regulatory Authority (Sahpra), which does not publish pending applications. Medical schemes such as Momentum exclude unregistered medicines, which doctors can obtain only with special Sahpra permission under section 21 of the Medicines Act. Obesity is not a prescribed minimum benefit, so schemes need not cover weight-loss jabs. Casgevy, at US$2.2-million in the US, remains out of reach in Africa.
South Africans are taking part, though. The University of Cape Town’s Lung Institute was a site for the lonvo-z trial, “a ground-breaking first for South Africa and the African continent”, and centres in Johannesburg, Pretoria and Cape Town joined the melanoma vaccine trial. More than 30 South Africans have received haemophilia gene therapies through trials, and UCT’s H3D drug discovery centre works with AI developers to tailor malaria and tuberculosis drug doses for African patients. – (c) 2026 NewsCentral Media
