At the annual meeting of the American Society of Clinical Oncology in May, results of a new drug for pancreatic cancer were presented, causing rare 42-second ovations. This success is part of a series of medical advancements this year, many of which are based on gene editing and artificial intelligence but remain in specialized press.
User X under the pseudonym Mgoes seeks to change this situation. On October 27, he published a post listing 10 such achievements, noting that the news 'is in press releases, and almost no one reads them.' Two days later, Elon Musk shared this post, stating that 'if you look closely enough, breakthroughs in everything are accelerating exponentially. An exponent always starts with something small.'
This account is pseudonymous and does not indicate scientific qualifications, but describes the owner as an investor in the venture fund 'SuperHuman Fund,' which focuses on 'human enhancement' and co-hosts the podcast Accelerate Bio, and has invested in some of the guests' companies.
Not all items on the list are breakthroughs, and only about half rely on gene editing or AI. However, collectively they demonstrate how these technologies are beginning to reach patients.
Gene editing alters the DNA instructions inside cells. The CRISPR-Cas9 technology, which earned Emma Sharpentie and Jennifer Doudna the Nobel Prize in Chemistry in 2020, became a medicine at the end of 2023 after regulators in the UK and US approved Casgevy for sickle cell anemia treatment. A newer version, known as base editing, works more like correcting a typo than cutting out an entire sentence.
Demos Hassabis and John Jumper from Google DeepMind received half of the 2024 Nobel Prize in Chemistry for AlphaFold, which predicts the structure of nearly all 200 million known proteins, helping scientists understand their functions. Artificial intelligence is now also used for selecting drug targets and developing molecules aimed at them.
This Year's Achievements
One result is a pill that helps extend the life of people with advanced pancreatic cancer. The drug daraxonrasib from Revolution Medicines approximately doubled the median survival time in a study involving 500 patients whose cancer had spread despite previous treatment: 13.2 months versus 6.7 months with chemotherapy. In August, it was approved by the U.S. Food and Drug Administration (FDA) under the name Rasonque. It blocks defective RAS proteins, which, according to the company, cause over 90% of pancreatic cancer cases.
Another achievement was a single gene-editing infusion preventing most episodes of painful swelling. In April, Intellia Therapeutics reported that a single administration of its CRISPR drug lonvo-z reduced episodes of hereditary angioedema by 87% compared to placebo. It edits a gene within the body to reduce the production of the protein causing the swelling, and 62% of patients had no episodes and did not require other therapy for six months. However, the procedure carries risks: US regulators suspended another internal treatment trial by Intellia after a patient developed severe liver damage and subsequently died.
A single treatment to lower cholesterol, potentially lifelong, was also developed. Researchers from Eli Lilly's subsidiary, Verve Therapeutics, reported that VERVE-102, which uses base editing to switch off the PCSK9 liver gene, reduced 'bad' cholesterol levels by 62% at the highest dose in an early study involving 35 people. The reduction was maintained for up to 18 months, although the longest observation period so far relates to patients on lower doses.
A new generation of weight loss injections has appeared. In May, Lilly announced that its weekly injection retatrutide helped people taking the maximum dose lose an average of 25% of their body weight over 80 weeks, including everyone who started treatment, although 11.3% on this dose discontinued due to side effects. This is a traditional drug mimicking three appetite and blood sugar hormones, unlike Ozempic, which mimics one.
AI helped find and design a drug for lung treatment. In September, Insilico Medicine began testing rentosertib in late-stage (Phase 3) trials in China for idiopathic pulmonary fibrosis—a disease that gradually causes lung scarring. AI analyzed biological data, identifying the TNIK enzyme as a target, and generative AI developed a drug to block it. The lead researcher of the trial, Professor Zuojun Xu, stated in an Insilico message that 'TNIK, the target identified by AI, has never been linked to fibrosis before.'
A vaccine was created to treat one patient's cancer. Computer programs identify up to 34 mutated targets unique to each patient's tumor, and the mRNA vaccine trains the immune system to search for them. In October, Merck and Moderna reported that their vaccine, when used with the immunotherapy drug Keytruda, reduced the risk of melanoma recurrence or death by 42% compared to Keytruda alone in a study of 1137 patients. It reduced the risk of cancer spread or death by 38%, less than 59% in an earlier, smaller study.
A drug to stimulate hair growth also emerged. In October, Veradermics announced that its pill met all goals in the second late-stage trial in men. This is minoxidil, a blood pressure control drug underlying Rogaine, repurposed into a slow-release tablet.
For some types of cancer, there is now the possibility of treatment without surgery. In October, the FDA granted preliminary, conditional approval for GSK's dostarlimab for rectal cancer, whose numerous genetic errors facilitate the immune system's detection of the tumor after the drug removes the brakes. Of the 154 patients in the main study, 71% received dostarlimab alone, requiring no surgery or other treatment, and showed no cancer progression.
Pig kidney as a bridge to transplantation. Tim Andrews lived a record 271 days without dialysis on a pig kidney containing 69 genetic edits until it was removed in October 2025 due to decreased function. In January, he received a human kidney, and The Lancet published this case, which his hospital describes as the first use of a pig kidney as a bridge to human transplantation.
DNA-exchanging cells. This is fundamental science, not medicine. A study published in the journal Cell in May showed the transfer of DNA fragments between cells through tiny tubes, carrying traits to recipient cells. However, this was laboratory work: researchers mixed two types of cells and introduced errors or damage to their DNA. It is unknown if this occurs in the body.
Results for pancreatic cancer, edema, cholesterol, and weight loss were published in the New England Journal of Medicine, while rectal cancer results were published in Annals of Oncology. Only the hair loss results are based exclusively on company data. Many trials are in early, small, or uncontrolled stages, and only two treatments have received approval.
The Start of Exponential Growth
An analysis conducted by Boston Consulting Group researchers in 2024 counted 75 AI-discovered molecules that entered clinical trials in humans between 2015 and 2023; this number grew by more than 60% annually. They passed initial safety tests in 80–90% of cases, which is 'significantly higher than historical industry averages.' However, in larger Phase 2 trials testing efficacy, only four out of ten were successful, which 'matches historical industry averages.'
No drug with both an AI-discovered target and an AI-designed molecule has yet been approved. Xu expects that rentosertib will require three to four years from the start of Phase 3 to receive approval 'under favorable conditions.'
Gene editing has progressed faster: from the first approved therapy Casgevy to the successful late-stage trial of internal editing lonvo-z in less than two and a half years. For now, the exponential growth Musk spoke of is evident in the number of new treatments entering trials, not yet in the number reaching patients.
Significance for South Africa
There are no records indicating that any of the 10 treatments are registered with the South African Health Products Regulatory Authority (Sahpra), which does not publish pending applications. Medical schemes, such as Momentum, exclude unregistered medicines that doctors can only obtain with special permission from Sahpra under Section 21 of the Medicines Act. Obesity is not a mandatory minimum benefit, so schemes are not obliged to cover weight loss injections. Casgevy, costing $2.2 million in the US, remains inaccessible in Africa.
Nevertheless, South Africans are participating in these processes. The University of Cape Town's Institute of Respiratory Medicine was a site for the lonvo-z trial, which was a 'groundbreaking first for South Africa and the African continent,' and centers in Johannesburg, Pretoria, and Cape Town participated in the melanoma vaccine trial. More than 30 South Africans underwent gene therapy for hemophilia in trials, and the H3D drug discovery center at UCT collaborates with AI developers to adapt malaria and tuberculosis drug dosages for African patients.
