Genespire patched together a €46.6 million (about $52 million) funding round to get its lead gene therapy into the clinic in a few years.
The Milan-based biotech, which employs nine people, said Wednesday that the Series B will fuel its mission to create a lentiviral vector-based gene therapy for children with methylmalonic acidemia, a rare genetic disease marked by disruptions to the breakdown of certain fats and amino acids. Also known as MMA, it can lead to muscular weakness, seizures, developmental delays, organ damage and even death.
Other gene therapy startups have attempted but then abandoned efforts to develop treatments for the disease, including
AstraZeneca’s
LogicBio
and
Selecta Biosciences
. There are no disease-modifying drugs, and treatment is currently addressed by diet modifications or sometimes liver transplants.
Genespire’s work is based on research out of the San Raffaele Telethon Institute for Gene Therapy. The four-year-old biotech recently recruited
former Lysogene CEO
Karen Aiach-Pignet as its new chief executive after Lysogene shut down.
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 said in an interview. “Probably more downs over the recent years.”
Several gene therapy makers, including
bluebird bio
— which saw its sickle cell gene therapy Lyfgenia get approved last year — are struggling after interest in the once-bustling field has waned amid the broader biotech downturn and questions about the profitability of making these therapies. That said, new biotechs are still emerging, with plans to focus on
neurodegenerative
and
CNS diseases
,
cancers
and other areas.
Aiach-Pignet joined Genespire after bringing Lysogene from the lab to Phase 3 with a gene therapy that
missed the bar
on improving cognitive development in Sanfilippo syndrome type A, a serious inherited neurodegenerative lysosomal storage disorder. Aiach-Pignet said a patient group called Blue Daisy has acquired the program.
Sofinnova Partners, which had backed Lysogene, co-led Genespire’s Series B. XGEN Venture, CDP Venture Capital and Indaco Venture Partners SGR are also investors in Genespire.
Aiach-Pignet said it is too soon to commit to a time frame for the Phase 1/2 study, though it is likely more than a year away, she said. The company still needs to coordinate the study with regulators and patient advocacy groups, according to the CEO.
“Something that I really pay great attention to is the viewpoints, the experience, the expertise of patient groups,” Aiach-Pignet said. “The reason why I wanted to include them in the process is because we need to have a very robust clinical protocol, but we also need to have a protocol that makes sense for the patients and will make sense for the payers down the line.”
Moderna is aiming for approval of its own experimental medicine for MMA by 2028. The biotech is in
Phase 1/2
with an
mRNA infusion
treatment candidate known as mRNA-3705. Meanwhile, a BridgeBio subsidiary called CoA Therapeutics
terminated
a Phase 1 of its oral drug
BBP-671
last year.
Editor’s note: This story was updated to include additional information on Blue Daisy acquiring Lysogene’s gene therapy.