New Partnership Offers Hope for Inherited Retinal Disease Therapies

Théa Acquires Key Inherited Retinal Disease Programs, Advancing Potential Treatments for LCA10 and Usher Syndrome

For individuals and families navigating the challenges of inherited retinal diseases (IRDs), news of advancements in therapeutic development offers a beacon of hope. A recent agreement between ProQR Therapeutics and Laboratoires Théa has solidified the future of two promising RNA-based therapies aimed at Leber congenital amaurosis 10 (LCA10) and Usher syndrome type 2a, bringing these potential treatments closer to the IRD community. This partnership means continued development for therapies that could one day make a significant difference in preserving and restoring vision.

Under the terms of the revised agreement, European eye care company Laboratoires Théa has acquired the ophthalmology assets from ProQR Therapeutics. This includes the rights to sepofarsen, a therapy designed for Leber congenital amaurosis 10 (LCA10), and ultevursen, intended to treat vision loss associated with Usher syndrome type 2a and non-syndromic retinitis pigmentosa. The deal involves an initial payment of €8 million (approximately $8.6 million), with potential for ProQR to receive up to €165 million (around $177 million) in additional payments based on development, regulatory, and commercial milestones, plus royalties on sales.

This acquisition follows an earlier attempt at a deal that did not materialize. However, both companies have now successfully finalized this new pact. ProQR had previously halted trials for sepofarsen and ultevursen and shifted its focus away from genetic eye diseases, making this acquisition by Théa crucial for the continued advancement of these programs.

For patients and families, this development signifies that research and development for these specific IRDs will continue under Théa's stewardship. Sepofarsen is an RNA antisense therapy targeting LCA10, a severe genetic disorder causing significant vision loss from early childhood. Ultevursen is another RNA-based therapy, aimed at addressing a specific mutation in the USH2A gene that causes retinitis pigmentosa and, in some cases, hearing loss, affecting thousands of patients in Europe and the United States. These antisense oligonucleotide (AON) therapies work by masking disease mutations in RNA, allowing cells to produce the correct protein. This approach is particularly beneficial for large retinal disease genes like CEP290 (LCA10) and USH2A (Usher syndrome), where gene replacement therapies can be challenging due to size limitations.

While sepofarsen previously showed meaningful vision improvements in some patients during ProQR's Phase 2/3 trial, it did not meet its primary or secondary endpoints in that study. Despite this, the Foundation Fighting Blindness expressed delight at the acquisition, noting that both emerging therapies have shown encouraging results in human studies and offer hope for preserving and restoring vision. Théa has a dedicated business unit, Sepul Bio, focused on these treatments and is committed to advancing them.

This partnership ensures that these potential treatments for LCA10 and Usher syndrome type 2a remain in active development, offering continued hope for the IRD community as they progress through clinical stages. The commitment from Laboratoires Théa to these programs underscores the ongoing dedication within the biotech industry to address unmet needs in inherited retinal diseases.