Inside Chiesi’s rare disease partnership playbook
In the dynamic and high-stakes world of rare disease innovation, partnerships between established pharmaceutical companies and early-stage biotech firms are essential to advancing transformative therapies. Giacomo Chiesi, the head of global rare diseases at Chiesi Group, said when it comes to early-stage biotech partnerships the firm begins with a patient-centric question: does the technology have the potential to meaningfully impact the lives of patients with rare diseases?
“While innovative modalities are exciting, our focus is always on the unmet patient need.”
“We evaluate the scientific rigor of the data, the manufacturability of the platform, and the potential for meaningful clinical impact,” he continued. “For example, in our collaboration with Key2Brain, which focuses on crossing the blood-brain barrier, we’ve prioritized the strength of the data and the partner’s expertise. The deal structure is designed to give the technology room to mature and de-risk over time while ensuring alignment on the mission to deliver meaningful therapeutic options to patients.”
With over $3 billion in deal experience, Chiesi said it has developed a balanced approach to its partnership portfolio to support both established platforms and next-generation technologies.
“In some conditions, proven platforms remain essential, while in others, such as genetically driven or metabolic diseases, newer technologies may offer transformative potential,” he told us. Chiesi said the firm’s partnership with Arbor reflects this approach.
“For primary hyperoxaluria type 1 (PH1), the burden of lifelong therapy is substantial, and exploring a potentially one-time gene editing option is an important step toward addressing this need. By grounding our decisions in patient experience and unmet need, we naturally maintain a portfolio that balances innovation with reliability.”
The challenges
Ultra-rare diseases present unique challenges as they often affect children and young adults. Chiesi described how its due diligence process reflects this reality.
“Scientific clarity, strong mechanistic rationale, and scalable manufacturing strategies are critical,” Chiesi said. However, he also placed emphasis on the patient-centric view.
“We also evaluate how a therapy fits into the lived experience of families. A treatment must alleviate burden, not add to it. Commercial viability in ultra-rare conditions depends on building a complete ecosystem – early diagnosis, centers of excellence, global access pathways, and thoughtful long-term follow-up.”
Chiesi told us that the company’s base in Boston, Massachusetts, has been “instrumental” in fostering partnerships. He hailed the location as an enabler to advancing “genetic medicine approaches alongside the scientific and patient communities they aim to serve.”
While the company has a base in Boston, the Italian family-owned pharma company is headquartered in Parma, Italy. Chiesi emphasized how it actively evaluates opportunities across Europe, the US, and other geographies.
“Regional partnerships can be highly valuable when they align with patient needs and offer opportunities to accelerate development or access within specific healthcare systems. Ultimately, what guides our decisions is the quality of the science, alignment with our rare disease strategy, and the potential to deliver tangible impact for patients,” he said.
Choosing the right partnership structures
Chiesi’s approach to partnership structures is also guided by the needs of the patient community and the strategic fit with its portfolio.
“The structure of our deals is built around what is safe, effective, and capable of advancing care,” he commented. When it comes to early-stage assets, the firm often favors staged or option-based collaborations.
“These allow both parties to learn from emerging data while managing risk,” Chiesi explained. “As programs mature, licensing, co-development, or acquisitions can provide greater clarity and speed.”
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