【Pharmaceutical Network Industry Dynamics】Cell and gene therapy drugs are a new track that major developed countries and economies around the world are competing to lay out, following small molecule drugs and antibody drugs. According to statistics, in 2025, there have been 9 major BD (including acquisitions) events in the gene therapy field, with a potential total amount exceeding 6.2 billion US dollars, involving pharmaceutical companies such as Eli Lilly, Chiesi, and Regeneron.
In June 2025, Eli Lilly and Company announced that it would acquire Verve Therapeutics, Inc. for up to $1.3 billion. This transaction aims to enhance Lilly's cardiovascular disease treatment portfolio through Verve's gene-editing therapies.
It is reported that Verve Therapeutics is committed to developing prevention and treatment methods for cardiovascular diseases through base editing technology, achieving permanent prevention of heart disease with a single injection. The company announced positive data from the Phase Ib clinical trial of its base editing therapy, VERVE-102. This therapy uses lipid nanoparticles (LNP) to deliver a Base Editor, permanently shutting down the expression of the PCSK9 gene in the liver, thereby reducing low-density lipoprotein cholesterol (LDL-C) levels to prevent and treat cardiovascular diseases, including heterozygous familial hypercholesterolemia (HeFH).
In October 2025, Italy's Chiesi Group and Arbor Biotechnologies announced an exclusive global collaboration and licensing agreement worth over $2 billion. The core of the collaboration is ABO-101, a clinical-stage new drug developed by Arbor for the treatment of Primary Hyperoxaluria Type 1 (PH1), as well as the multi-target selection for developing novel liver-targeted therapies using Arbor’s gene-editing platform.
Reportedly, ABO-101 is a one-time treatment drug specifically targeting the HAO1 gene in the liver, inhibiting oxalate production at its source and thereby preventing the accumulation of those fatal crystals. Currently, Arbor has initiated the Phase I/II clinical trial of ABO-101, named redePHine, planning to recruit 23 patients with the main trial expected to conclude by March 2029. According to the cooperation agreement, Chiesi will collaborate with Arbor to advance this trial going forward.
In October 2025, AviadoBio and UgeneX entered into an exclusive option and license agreement for the development and commercialization of the optogenetics pipeline UGX-202. UGX-202 is an adeno-associated virus (AAV) gene therapy currently in clinical development for the treatment of patients with retinitis pigmentosa (RP). A second undisclosed indication is expected to enter the clinical stage by the end of the year.
In December 2025, Regeneron Pharmaceuticals and Tessera Therapeutics reached a collaboration on gene-editing drugs for alpha-1 antitrypsin deficiency, with the total value of the collaboration reaching $275 million. Industry experts noted that the significant investment by Regeneron and numerous biotech companies in alpha-1 antitrypsin deficiency is partly due to unmet clinical needs and the clear logic behind gene therapy. This is an autosomal codominant genetic disorder primarily caused by mutations in the SERPINA1 gene, which encodes alpha-1 antitrypsin—a protein mainly synthesized by the liver and responsible for protecting lung tissue from excessive degradation by neutrophil elastase.
The BD boom in the gene therapy field in 2025, at its core, is the result of the industry's development reaching a critical stage. Multinational pharmaceutical companies are rapidly acquiring innovative technologies and pipelines through mergers and acquisitions, while startups are realizing value through their core technologies, forming a "leading + emerging" synergistic development model. In the future, with continued investment from more leading companies, along with technological iteration and industry maturation, gene therapy drugs will truly enter accessible healthcare, ushering in a new era of biomedicine.
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