The ambition behind GLP-1 halo: Lilly invests billions more to lay out the next track
On August 7, 2025, Eli Lilly's semi annual financial report once again caught the attention of the industry: total revenue was 28.286 billion US dollars, a year-on-year increase of 41%. Among them, the "weight loss miracle drug" Tilpotide sold 14.734 billion US dollars in the first half of the year alone, accounting for more than half of the total revenue.
But under the halo of this GLP-1 "star drug", a larger layout has quietly been pushed forward - Lilly is making gene therapy the next core of growth.
The turn of Lilly actually had a foreshadowing long ago.
In 2024, out of the total revenue of $45.043 billion, the cardiovascular metabolism field contributed $29.521 billion, accounting for a staggering 66%.
The risk of relying too much on a single field has made the management determined to launch a 'post GLP-1' strategy.
Dr. Dan Skovronsky, Executive Vice President of the company, put it bluntly: "Almost all major illnesses stem from genetic mutations, and gene therapy is the true path to curing diseases in the future
The global central nervous system drug market is expected to reach $172.1 billion by 2034, but currently, Lilly's neuroscience segment only accounts for 3% of total revenue.
1.This business imbalance has become a key driving force for betting on gene therapy.
Especially for neurodegenerative diseases such as Alzheimer's disease and Parkinson's disease, existing therapies can at most alleviate symptoms, while gene therapy has the potential to repair lesions from the root.
The biggest difference between gene therapy and traditional drugs is that it directly targets the root of the disease - genetic defects.
Taking Parkinson's disease as an example, 5% -10% of patients carry GBA1 gene mutations, which result in the loss of function of key enzymes.
The GBA1 gene therapy developed by Eli Lilly uses viral vectors to deliver normal genes into patient cells, restoring enzyme function and delaying neuronal degeneration.
This' reparative therapy 'completely rewrites the logic of traditional medicine's' palliative therapy'.
The GRN gene therapy under development by Eli Lilly targets frontotemporal dementia and can repair mutated genes through gene editing; OTOF gene therapy has even shown the possibility of restoring hearing in clinical trials.
These cases illustrate that gene therapy is gradually expanding from the field of rare diseases to more common mass diseases.
2、 Lilly's strategy is to turn gene therapy into a 'super engine', and Lilly's strategy is very clear:
In April 2025, the company spent $1.4 billion to introduce Sangamo's STAC-BBB technology. This new adenovirus capsid has a 700 fold higher efficiency in penetrating the blood-brain barrier than AAV9 in non-human primate experiments, providing a breakthrough solution for drug delivery in neurodegenerative diseases.
In the field of RNA editing, the trans splicing ribozyme technology developed by Eli Lilly and Rznomics can avoid the risk of permanent genome changes;
In terms of CRISPR gene editing, the VERVE-102 therapy obtained after acquiring Verve Therapeutics can permanently shut down the expression of PCSK9 gene in the liver with one infusion, reducing low-density lipoprotein cholesterol by 53% -69%. It may completely change the treatment of cardiovascular diseases.
What Novo Nordisk missed was precisely what Eli Lilly caught
Next, in 2022, Lilly spent $610 million to acquire Akouos, a company specializing in self research, cooperation, and acquisition, officially entering the field of gene therapy for hearing loss;
The Boston Gene Medicine R&D Center, which will be opened in 2024, has become an innovation base for 700 scientists - two-thirds of the space has been given to laboratories, and a "Lilly Gateway Labs" incubator has been established, bringing in four cutting-edge biotech companies including Amplitude Therapeutics.
This "self-developed+external cooperation+strategic acquisition" model makes Lilly particularly flexible in adjusting its pipeline.
For example, when the Duchenne muscular dystrophy therapy previously collaborated with Precision BioSciences turned yellow, it immediately shifted to other more promising projects. The sales revenue of 14.7 billion US dollars in the first half of the year for Tilpotide has accumulated a solid "family base" for Eli Lilly.
This' cash cow 'effect gives it more confidence to invest in gene therapy.
3、 The future direction can be seen from Lilly's layout, which includes three major directions for gene therapy:
From a rare disease to a common disease, Lilly's Lepidiriran therapy for atherosclerotic cardiovascular disease reduced the Lp (a) level by 93.9% on average in the second phase study.
Once this gene therapy for diseases with high incidence rate becomes available, the research and development cost can be reduced a lot, and the price may be more user-friendly.
The single infusion mode of lifelong VERVE-102 therapy may be able to transform the treatment of cardiovascular diseases from long-term medication to a "one-time solution". For long-term diseases such as diabetes and chronic pain, this is a subversive change.
Earning 126.4 billion in just six months! Simeglutide kicks K drug and firmly sits on the throne of global medicine king
We have built a technology platform and ecological ecosystem that links AAV vectors, RNA editing, gene regulation and other technologies together, creating a complete gene therapy ecosystem.
This systematic layout has increased its chances of winning against giants such as Novartis and Roche. Ultimately, Lilly is transitioning from a traditional pharmaceutical giant to an innovator in genetic medicine.
With the full operation of the Boston R&D center and the advancement of more than ten clinical pipelines, gene therapy is likely to become its next billion dollar growth engine.
As Dr. Skovronsky said, "We are paving the way for a future beyond Alzheimer's disease and obesity
When gene therapy truly enters large-scale clinical practice from the laboratory, this bold gamble by Eli Lilly may truly rewrite the global medical landscape.
