Ambition beyond the halo: Lilly invests another ten billion to lay out the next track

Lilly's shift was actually foreshadowed long ago.

In 2024, the company's total revenue amounted to $45.043 billion, of which the cardiovascular and metabolic field contributed $29.521 billion, accounting for a whopping 66%.

The risk of relying too heavily on a single field has compelled the management to resolutely launch a "post-GLP-1" strategy.

Dr. Dan Skovronsky, the executive vice president of the company, put it bluntly: "Almost all major diseases stem from genetic mutations, and gene therapy is the path towards truly curing diseases in the future."

The global central nervous system drug market is expected to reach $172.1 billion by 2034, but currently, Eli Lilly's neuroscience segment only accounts for 3% of its total revenue.

This imbalance in business 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 best alleviate symptoms, while gene therapy holds promise for fundamentally repairing the lesions.

The biggest difference between gene therapy and traditional drugs lies in that gene therapy directly targets the root cause of the disease - genetic defects.

In the case of Parkinson's disease, 5%-10% of patients carry mutations in the GBA1 gene, which results in the loss of function of a key enzyme.

The GBA1 gene therapy developed by Eli Lilly utilizes viral vectors to deliver normal genes into patients' cells, restoring the function of enzymes and thereby delaying neuronal degeneration.

This "restorative treatment" completely overturns the logic of traditional drug "palliative treatment".

Lilly's GRN gene therapy, which is under development, targets frontotemporal dementia and can repair mutated genes through gene editing; OTOF gene therapy has even shown potential in clinical trials to restore hearing.

These cases all illustrate that gene therapy is gradually expanding from the field of rare diseases to more common diseases affecting the general population.

Second, Eli Lilly's approach is to turn gene therapy into a "super engine". Eli Lilly's approach is very clear:

Technological integration first In April 2025, the company invested $1.4 billion to introduce Sangamo's STAC-BBB technology, a novel adeno-associated virus capsid. 

In non-human primate trials, it demonstrated a 700-fold higher efficiency in penetrating the blood-brain barrier compared to AAV9, providing a breakthrough solution for drug delivery in neurodegenerative diseases.

In the field of RNA editing, the trans-splicing ribozyme technology collaboratively developed by Eli Lilly and Rznomics can avoid the risk of permanent genomic changes;

In terms of CRISPR gene editing, the VERVE-102 therapy, acquired after the purchase of Verve Therapeutics, can permanently shut down the expression of the PCSK9 gene in the liver with a single infusion, reducing low-density lipoprotein cholesterol by 53%-69%. 

It may completely change the treatment method for cardiovascular diseases.

What Novo Nordisk missed, Eli Lilly seized

Next, focusing on self-research, cooperation, and acquisition. In 2022, Eli Lilly spent $610 million to acquire Akouos, officially entering the field of gene therapy for hearing loss;

The Boston Genomics Medical Research and Development Center, which opened in 2024, has become an innovation hub for 700 scientists. 

Two-thirds of the space is dedicated to laboratories, and an "Lilly Gateway Labs" incubator has been established, attracting four cutting-edge biotech companies such as Amplitude Therapeutics to settle in.

This model of "in-house R&D + external collaboration + strategic acquisition" provides Eli Lilly with exceptional flexibility in adjusting its product pipeline.

For example, the Duchenne muscular dystrophy therapy previously collaborated with Precision BioSciences fell through, and the company immediately shifted to other more promising projects. 

Meanwhile, the sales revenue of $14.7 billion from TACOLIN in the first half of the year has accumulated a solid "financial foundation" for Eli Lilly.

This "cash cow" effect gives it more confidence to invest in gene therapy.

III. Future Directions: From Eli Lilly's layout, we can see three major directions for gene therapy:

From rare disease to common disease: Lilly's Lepodisiran therapy for atherosclerotic cardiovascular diseases achieved an average reduction of 93.9% in Lp(a) levels in its Phase II study. 

This gene therapy, targeting a disease with a high incidence rate, could significantly reduce research and development costs once successful, potentially making the price more affordable.

One treatment for life-long management: The single infusion model of VERVE-102 therapy may transform the treatment of cardiovascular diseases from long-term medication to a "one-time solution." For conditions requiring long-term management such as diabetes and chronic pain, this is a revolutionary change.

Building a technology platform and ecosystem: Eli Lilly has integrated technologies such as AAV vectors, RNA editing, and gene regulation to establish a comprehensive gene therapy ecosystem.

This systematic layout has given it a significant advantage when competing with giants like Novartis and Roche. Ultimately, Eli Lilly is transitioning from a traditional pharmaceutical giant to a "genetic medicine innovator".

With the full operation of the Boston R&D center and the advancement of over ten clinical pipelines, gene therapy is likely to become its next billion-dollar growth engine.

As Dr. Skovronsky said, "We are laying the groundwork for a future beyond Alzheimer's disease and obesity."

Once gene therapy moves from the laboratory to large-scale clinical trials, Lilly's bold gamble may indeed reshape the global healthcare landscape.