Italian Gene Therapy Startup Secures $52M for Rare Genetic Disease Targeted by Moderna

Genespire has successfully secured €46.6 million (approximately $52 million) in a Series B funding round, aiming to advance its leading gene therapy candidate into clinical trials within the coming years. This biotech company, based in Milan and comprising a small team of nine employees, is focused on developing a lentiviral vector-based gene therapy to treat methylmalonic acidemia (MMA). MMA is a rare genetic disorder that disrupts the metabolism of certain fats and amino acids, potentially causing severe complications such as muscular weakness, seizures, developmental delays, organ damage, and even death.

The field of gene therapy for MMA has seen multiple efforts, yet companies like AstraZeneca’s LogicBio and Selecta Biosciences have eventually abandoned their pursuits. Presently, there are no treatments available that modify the disease; management of MMA largely relies on dietary adjustments and, in some cases, liver transplants.

Genespire's initiative is grounded in research conducted at the San Raffaele Telethon Institute for Gene Therapy. The company, which started four years ago, recently appointed Karen Aiach-Pignet as its new CEO. Aiach-Pignet previously served as CEO of Lysogene, a company that was forced to shut down. In an interview, she expressed cautious optimism about her new role, acknowledging the recent struggles within the gene therapy sector. “It’s a new page for me at a time when there have been a lot of ups and downs in the gene therapy space,” she noted.

The gene therapy industry has faced several challenges in recent years. Although companies like bluebird bio achieved milestones—such as the approval of their sickle cell gene therapy Lyfgenia last year—the overall interest in the field has diminished. This decline is attributed to the broader downturn in the biotech industry and concerns regarding the economic viability of these therapies. Despite these challenges, new biotech companies continue to emerge, targeting diseases such as neurodegenerative and CNS conditions, cancers, and other critical areas.

Aiach-Pignet brings a wealth of experience to Genespire, having led Lysogene through its development phases to a Phase 3 trial. Lysogene’s gene therapy for Sanfilippo syndrome type A, a severe inherited neurodegenerative disorder, did not meet the necessary benchmarks for improving cognitive development. The program was recently acquired by a patient group named Blue Daisy.

The Series B funding round for Genespire was co-led by Sofinnova Partners, which was also an investor in Lysogene. Other participants in the funding round included XGEN Venture, CDP Venture Capital, and Indaco Venture Partners SGR.

As for the timeline of the upcoming clinical trials, it remains uncertain. Aiach-Pignet mentioned that starting a Phase 1/2 study is likely more than a year away. The company needs to coordinate the trial with regulatory authorities and patient advocacy groups. She emphasized the importance of including patient groups in the process to develop a robust and patient-centric clinical protocol, which would also be appealing to payers in the future.

Meanwhile, other companies are also striving to develop treatments for MMA. Moderna is targeting approval for its experimental MMA medicine by 2028. Their candidate, mRNA-3705, is currently in Phase 1/2 clinical trials. On the other hand, CoA Therapeutics, a subsidiary of BridgeBio, terminated its Phase 1 trial for the oral drug BBP-671 last year.

Genespire’s advancement in gene therapy for MMA represents a significant step forward, offering new hope for patients affected by this debilitating disease.