Genespire

Gene therapy fixes genetic defects causing diseases by introducing functional gene copies into patient cells to restore normal activity.

Gene therapy corrects genetic defects by transferring functional gene copies into patient cells to restore normal activity.

Genespire develops GENE202, an ISLV-based gene therapy, to treat patients with methylmalonic acidemia (MMA).

Genespire is developing GENE202, an ISLV-based gene therapy, to treat patients affected by methylmalonic acidemia (MMA).

Jörn Aldag serves as CEO of Hookipa Pharma, a clinical-stage biopharmaceutical company developing next-generation immunotherapies for infectious diseases and tumors using its proprietary arenavirus platform to reprogram the human immune system. Before joining Hookipa in June 2016, he led uniQure N.V., a NASDAQ-listed company pioneering gene therapy research with adeno-associated viruses.

Luigi Naldini directs the San Raffaele Telethon Institute for Gene Therapy and teaches at San Raffaele University in Milan, Italy. Over the past 30 years, he has led the development of lentiviral vectors for gene transfer, now widely used in biomedical research and in clinical trials for treating genetic diseases.

Luigi Naldini directs the San Raffaele Telethon Institute for Gene Therapy and teaches at San Raffaele University in Milan, Italy. For the past 30 years, he has pioneered the development and applications of lentiviral vectors for gene transfer, now widely used in biomedical research and clinical trials for treating genetic diseases.

Julianne Smith is Chief Development Officer at Genespire, bringing over 30 years of scientific research experience, including 18 years in biotech. Previously, she worked at Cellectis S.A., a global leader in gene editing and allogeneic genetically modified T-cell (UCART) therapies listed on Nasdaq, holding senior roles managing research teams developing genetically modified T-cell products.

Sofinnova Partners is a European venture capital firm specializing in life sciences, with offices in Paris, London, and Milan. The firm combines medical, scientific, and business expertise to support biotech companies from early to advanced stages of development.

Sofinnova Partners is a leading European venture capital firm in life sciences, specializing in healthcare and sustainability. Based in Paris, London, and Milan, the firm brings together global professionals with expertise in science, medicine, and business to support biotech companies across all development stages.

Genespire develops ready-to-use gene therapies. Its immune shielded lentiviral vectors (ISLV) enable permanent therapy production directly from the liver in a single treatment for adults and children with genetic diseases.

Genespire advances its single-dose gene therapy, **GENE202**, into clinical trials in 2026. The biotech firm specializes in rare disease treatments, targeting unmet medical needs with gene-editing approaches. Lucia Faccio, CEO and Sofinnova Partners partner, oversees the development milestone.

Jan 2026Clinical Trials ArenaNews article

Genespire secures Orphan Drug Designation from the FDA and European Commission for its gene therapy candidate **GENE202**, targeting **Methylmalonic Acidemia (MMA)**, a rare pediatric metabolic disorder with no existing approved treatments. The therapy uses an immune-shielded approach to address the unmet medical need in patients. Regulatory recognition accelerates development for this life-threatening condition affecting children.

Jan 2026packgeneNews article

Genespire presents preclinical dosing data for its first-in-human **in vivo liver-directed ISLV gene therapy** targeting **MMA** at the **ASGCT Annual Meeting**. The oral presentation highlights insights from preclinical studies, focusing on dosing strategies for the therapy. Genespire develops gene therapies for rare genetic diseases.

May 2025kommunikasjon.ntb.noNews article

Genespire raises €46 million to advance its pediatric gene therapy for methylmalonic acidemia (MMA) into clinical trials. The funding, one of Italy’s largest private biotech rounds, also supports expanding the company’s gene therapy development pipeline. The therapy targets a rare metabolic disorder affecting children.

Sep 2024ITTBioMedNews article