GSK and CAMP4 Forge Alliance to Boost Protein Levels in Genetic Disease

CAMBRIDGE, Mass. – December 18, 2025 – Pharmaceutical giant GSK is placing a significant bet on a novel gene-regulating technology, entering a strategic collaboration with clinical-stage biotech CAMP4 Therapeutics. The deal, centered on developing new therapies for challenging neurodegenerative and renal diseases, provides CAMP4 with $17.5 million upfront and validates its unique approach to treating genetic disorders.

The partnership will leverage CAMP4’s proprietary RAP Platform® to discover and develop antisense oligonucleotide (ASO) drug candidates. Unlike many existing RNA therapies that aim to silence or reduce harmful proteins, CAMP4’s technology is designed to do the opposite: upregulate, or increase, the expression of healthy proteins. GSK will take the lead on advancing any resulting drug candidates through further development and commercialization.

“Protein under-expression plays a critical role in diseases such as neurodegenerative and kidney disease," said Josh Mandel-Brehm, President and Chief Executive Officer of CAMP4. "Our collaboration with GSK, focused on the rapid identification of novel targets and potential ASO therapeutics that increase the expression of validated genetic targets, underscores the potential of our discovery platform to create transformational medicines for patients.”

In addition to the initial payment, CAMP4 is eligible for future milestone payments tied to development and commercial success, as well as tiered royalties on any eventual product sales.

A New Frontier in RNA Therapeutics

At the heart of the collaboration is CAMP4’s Regulatory RNA Activating Platform, or RAP Platform®. This technology represents a distinct and potentially groundbreaking approach within the booming field of RNA therapeutics. While pioneers like Ionis and Alnylam have built successful platforms primarily focused on silencing disease-causing genes, CAMP4 is charting a different course by targeting the cellular machinery that controls how much of a gene is expressed.

The platform hones in on a class of molecules known as regulatory RNAs (regRNAs). These molecules act as master switches, controlling the rate of gene transcription—the first step in creating a protein. By designing ASOs that specifically interact with these regRNAs, CAMP4 aims to fine-tune and amplify the production of essential proteins that are deficient in certain genetic diseases.

This approach is particularly promising for a large class of conditions known as haploinsufficient disorders, where individuals have one faulty copy and one healthy copy of a gene, leading to insufficient protein levels. The company estimates there are over 1,200 such diseases where even a modest boost in healthy protein could provide a significant therapeutic benefit. This ability to "turn up the dial" on gene expression, rather than just turning it off, opens a new therapeutic playbook for genetic medicine.

GSK's Strategic Play in a Competitive Field

For GSK, the deal is more than just an investment in a single platform; it's a calculated move to deepen its capabilities in the highly competitive RNA therapeutics space and reinvigorate its pipeline in complex disease areas. The company is actively building a diversified portfolio of RNA-targeting technologies, with recent deals spanning splice modulators and other ASO programs, including Bepirovirsen for chronic hepatitis B.

This collaboration allows GSK to tap into a cutting-edge discovery engine for a relatively modest upfront investment, mitigating the high risks and costs of early-stage research. By taking responsibility for later-stage development and commercialization, GSK can leverage its global scale and expertise to bring potential new medicines to market.

The partnership also aligns with GSK's renewed strategic focus on neuroscience. After scaling back in previous years, the company has been making a concerted push back into neurodegenerative disease research, an area with immense unmet medical need. This is evidenced by recent high-value licensing deals aimed at overcoming challenges like crossing the blood-brain barrier to deliver therapies for conditions such as Alzheimer's and Parkinson's disease.

Chris Austin, SVP Research Technologies, GSK, highlighted the synergy between the two companies. “We are excited to collaborate with CAMP4, combining their RNA discovery platform to increase specific gene activity with GSK’s expertise in therapeutic oligonucleotides, genetics and advanced laboratory and data technologies," he stated. "This agreement aims to drive the development of novel medicines for neurodegenerative and kidney disease and demonstrates our approach of harnessing cutting-edge technologies to deliver transformational therapies for patients.”

Hope on the Horizon for Intractable Diseases

The collaboration's focus on neurodegenerative and kidney diseases targets conditions that have historically proven difficult to treat. For countless patients and families, current therapies often only manage symptoms or slow progression, with few options available that address the underlying genetic cause.

The field of ASO therapeutics has already delivered paradigm-shifting results, offering a blueprint for what might be possible. The success of drugs like nusinersen (Spinraza) for spinal muscular atrophy (SMA) and tofersen (Qalsody) for a genetic form of amyotrophic lateral sclerosis (ALS) has proven that RNA-targeted drugs can fundamentally alter the course of devastating neurological disorders.

By applying a novel upregulation mechanism, the CAMP4-GSK partnership hopes to extend this success to a new set of diseases. In neurodegeneration, many conditions are linked to insufficient levels of critical proteins. Similarly, numerous genetic kidney diseases are caused by a partial loss-of-function in key genes. A therapy capable of restoring protein levels towards a normal range could offer a disease-modifying treatment where none currently exists. The ASO market is projected to expand dramatically, with some analysts predicting it could exceed $10 billion by 2030, largely driven by its potential in neurological and rare genetic disorders.

This strategic alliance between a nimble biotech innovator and a pharmaceutical powerhouse illustrates a prevailing trend in modern drug development. While CAMP4 provides the specialized discovery engine, GSK brings the resources and global infrastructure needed to navigate the long and expensive path through clinical trials, regulatory approval, and worldwide distribution, potentially accelerating the journey of these next-generation therapies from the laboratory to patients in need.