GeneSpire Srl

Italian VC XGEN Venture plans to make nine more investments within the next three years.\n Italian VC XGEN Venture has closed a 180 million euro ($190 million) fund for biotech and medtech companies.The life sciences fund is XGEN’s first, and the money will go toward early-stage businesses, according to a Dec. 18 release.  The European VC has already started deploying money to six startups, including Italian gene therapy company Genespire’s $52 million series B and Merck KGaA cancer spinout iOnctura.The firm plans to make nine more investments within the next three years to round out the fund’s portfolio. “Our goal over the next years is to consolidate ourselves as one of the leading players in the European venture capital market,” XGEN co-founder and managing partner Daniele Scarinci said in the release. “The remarkable success of our first fund and the exceptional team built since inception provide a solid foundation for reaching such an ambitious target.”

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.

Genespire’s GENE202 was developed using the company’s Immune Shielded Lentiviral Vector (ISLV) platform that allows a patient’s liver to produce the therapy throughout its lifetime. Image credit: jack8 / Shutterstock. Italian biotech Genspire is set to launch a Phase I/II clinical trial of its lead gene therapy, GENE202, as a treatment for a group of rare, inherited diseases – methylmalonic acidemia. The company will be able to fund the trial after it received €46.6m ($52.1m) in a Series B financing round. The financing will also help to fund discovery and preclinical work for other gene therapy candidates. Investors included European investment firms such as Sofinnova Partners, XGEN Venture and CDP Venture Capital. Genspire is a spinout from Milan-based San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), and has previously raised €16m in Series A financing in 2020. Methylmalonic acidaemia is a group of rare genetic disorders where an individual is unable to metabolise proteins and fats properly. This results in high levels of acid in the blood (acidaemia) and body tissues. Methylmalonic acidaemia often manifests within the first few months of life and its severity can vary from mild disease to life-threatening. According to the US National Organization for Rare Disorders (NORD), methylmalonic acidaemia usually affects 1 in 50,000 to 1 in 100,000 live births. The disease is usually managed through dietary modifications to limit certain proteins and fats. See Also: Biologic API Manufacturing Companies (Protein and Peptide) in Contract Manufacturing for the Pharmaceutical Industry Bavarian Nordic gains US contract for additional mpox vaccine GENE202 is an off-the-shelf, one time gene therapy developed using the company’s Immune Shielded Lentiviral Vector (ISLV) platform. ISLV-based therapies are designed to allow a patient’s liver to produce the therapy throughout its lifetime. Gene therapies are a growing area of interest , with multiple gene therapies gaining approval from the US Food and Drug Administration (FDA) in recent years. In recent months, multiple companies have entered the gene therapy market by acquiring or merging with specialist companies and pipelines. Last year, Alexion AstraZeneca Rare Disease signed a definitive agreement, acquiring and licensing Pfizer ’s early-stage gene therapy portfolio for rare diseases . The deal included a $1bn payment along with net sale royalties from the acquired therapies. Despite the recent interest, the uptake of gene therapies is limited due to their high price tags . Furthermore, not all gene therapies make it to market, especially for rare diseases, as their high cost and limited commercial market can deter companies from investing. In some cases, academic institutes and non-profit organisations have ‘rescued’ these gene therapy programmes. Another therapy in development for methylmalonic acidaemia is Moderna ’s mRNA therapy, mRNA-3705. The therapy is being investigated in the Phase I/II Landmark study (NCT04899310) as a treatment for a specific type of methylmalonic acidaemia, one due to CoA mutase (MUT) deficiency. In June 2024, mRNA-3705 was included in a Support for Clinical Trials Advancing Rare Disease Therapeutics (START) pilot programme by the US Food and Drug Administration (FDA). The START programme is aimed at expediting the development of novel drugs and biological products for rare diseases. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva . Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content. Free Whitepaper Cell and gene therapies: Pipe dream to pipeline The cell and gene industry is gaining momentum, with a new wave of therapies promising to transform the way doctors treat, and even cure, disease. In this report, Cytiva and GlobalData have collaborated to explore the rise of the cell and gene therapy industries, the current state of the market, present and future opportunities for advancement, and the challenges that lie ahead. Thank you. You will receive an email shortly. Please check your inbox to download the Whitepaper. By Cytiva Thematic By downloading this Whitepaper, you acknowledge that GlobalData may share your information with Cytiva Thematic and that your personal data will be used as described in their Privacy Policy