In new deal, pharma company acquires DEB gene therapy developer
With Replay platform, Leo Pharma will advance topical gel for rare genetic disease
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In a new deal, Denmark-based Leo Pharma is acquiring Replay — and its “next-generation gene therapy platform” — with an eye toward developing a topical treatment for dystrophic epidermolysis bullosa (DEB), a rare genetic disease causing extremely fragile skin.
The gene therapy company, based in the U.S., is the developer of a gel based on the herpes simplex virus (HSV) that delivers a healthy gene into skin cells to treat DEB.
“Replay’s HSV gene therapy platform holds significant promise for patients with rare genetic skin diseases, and realizing its full potential requires focused expertise in medical dermatology — an area where Leo Pharma brings decades of leadership, scale and proven execution,” Christophe Bourdon, Leo’s CEO, said in a company press release announcing the deal.
All types of epidermolysis bullosa cause the skin to be very fragile, tearing easily when touched.
In dystrophic epidermolysis bullosa, this is due to mutations in the COL7A1 gene, which encodes part of type VII collagen, a protein that helps hold skin layers together. When it is faulty, even small movements can cause the layers to separate and form blisters.
Despite treatments, ‘significant unmet need’ in DEB
Existing DEB therapies include the cell therapy Zevaskyn (prademagene zamikeracel) and the gene therapy Vyjuvek (beremagene geperpavec), which was the first for this condition to be approved by the U.S. Food and Drug Administration.
Still, DEB “remains one of the most devastating genetic skin diseases with significant unmet need,” said Martin Steiner, managing director at DEBRA Research, a nonprofit dedicated to advancing therapies for this disease.
For Steiner, “pairing Replay’s HSV gene therapy platform with Leo Pharma’s dermatology expertise and global infrastructure is exactly the kind of combination the [dystrophic epidermolysis bullosa] community needs.”
“We welcome Leo Pharma’s commitment to rare disease and look forward to seeing this platform advance,” Steiner said.
Replay’s technology uses a modified HSV to deliver a healthy version of the COL7A1 gene into skin cells. One advantage of HSV is that it can carry large genes. Once COL7A1 enters skin cells, they can use it to produce the part of type VII collagen that was faulty or missing. This is expected to help the layers of skin hold together, promoting wound healing.
The gene therapy, now in preclinical evaluations to understand if it can be tested in patients, is being developed as a gel that can be applied directly to the skin. This topical approach means the gene therapy would work where it’s needed most.
Patients may also be able to receive repeated doses over time if needed, according to the developer.
“Natural tropism [specificity] for skin cells and the ability to redose patients make HSV uniquely suited to treating rare genetic dermatological conditions,” said Lachlan MacKinnon, Replay’s CEO. “Together with Leo Pharma’s decades of dermatology leadership and global infrastructure, purpose-built for skin diseases, we can bring therapies to the patients who need them most and build an enduring dermatology franchise in rare genetic medicine.”
Leo will combine its long experience in dermatology with Replay’s gene therapy expertise to move this work forward. The agreement includes an upfront payment of $50 million, as well as the promise of future payments if the gene therapy reaches certain development milestones. This would involved tiered single-digit royalties, the company said.
“Using our unique [artificial intelligence]‑powered scouting platform, Innoviewer, we identified Replay as a high‑potential opportunity. The acquisition aligns with our strategy of investing in the most impactful opportunities in dermatology and positions Leo Pharma at the forefront of next‑generation gene therapy,” Bourdon said.
