Lysogene SA

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.

PRESS RELEASE Genespire appoints Karen Aiach-Pignet as Chief Executive Officer Karen’s appointment bolsters Genespire’s position as a leader in in-vivo lentiviral gene therapy Milan, Italy, 12 September 2024 – Genespire, a biotechnology company developing off-the-shelf gene therapies for pediatric patients affected by genetic diseases, today announces the appointment of Karen Aiach-Pignet as Chief Executive Officer following Dr. Julia Berretta, the former CEO, stepping down. Karen has an outstanding track-record in gene therapy applied to genetic neurodegenerative diseases having founded and led Lysogene, a global leader in the field. During her tenure, she pioneered the development of an adeno-associated virus (AAV) vector for patients with the rare neurodegenerative condition Sanfilippo Type A, initiated and executed an international Phase 3 gene therapy clinical trial, and raised funds via partnering deals and by listing the company on the Euronext Stock Exchange. Karen joins Genespire as the company progresses lead candidate, GENE202, for the treatment of methylmalonic acidemia (MMA) into clinical development. MMA is a devastating genetic disorder impairing the metabolism of certain amino acids and fats. Onset occurs in early infancy, is associated with significant mortality and morbidity, and there are currently no disease-modifying treatments available. Genespire’s technology has the potential to help treat many other genetic diseases with a one-time, off-the-shelf treatment, for both adult and pediatric patients. Karen Aiach-Pignet, CEO at Genespire, commented: “I strongly believe in a better world for patients with metabolic diseases. Leading Genespire, with the aim to develop and deliver safe and efficacious life changing therapies for patients with substantial needs, is therefore a huge honour. Our unique ISLV technology platform, our investors, the team and I, will all come together to make this company a success. Embarking on this new partnership with Sofinnova Partners and working with other high-profile investors, provides huge endorsement for what we are aiming to achieve.” Joern Aldag, Chairman at Genespire, commented: “I am incredibly proud of what the Genespire team has achieved and would like to thank Dr. Julia Berretta, our previous CEO, for her excellent work over the past four and a half years and for orchestrating a seamless transition of leadership. We wish her all the best for the future. “Karen’s extensive experience and knowledge in the development of gene therapies for rare diseases and paediatric indications is a true inspiration. She has incredible drive, ambition and the ability to deliver fast results which will be an invaluable asset to Genespire as we progress our lead lentiviral-based therapy into the clinic.” In addition to her many prestigious accomplishments, Karen is a founding executive member of the International Rare Diseases Research Consortium, was a Member of the Paediatric Committee at the European Medicines Agency and served on the French Ethical Review Board at Ambroise Paré Hospital. She has supported several not-for-profit organizations engaged in advocacy and research in the field of rare diseases. Acknowledgements of Karen’s work include the Ordre National du Merite, a French order of merit, awarded by the French President. Karen recently studied Digital Health at Harvard Business School. She is a graduate from the ESSEC Business School and majored in Economics at the Institut d’Études Européennes of University Paris 8. -ENDS- Enquiries: Genespire Tel: +39 02 80896651 info@genespire.com ICR Consilium Tel: +44 (0) 20 3709 5700Amber Fennell/Ashley Tapp/Jonathan EdwardsGenespire@consilium-comms.com About GenespireGenespire is a biotechnology company, developing off-the-shelf gene therapies based on immune shielded lentiviral vectors (ISLVs) for pediatric patients affected by genetic diseases. ISLVs are designed to be used intravenously and allow the life-long production of the therapy directly from the patient’s liver. Genespire is initially advancing therapeutic programs in inherited metabolic diseases with high unmet medical need. Based in Milan, Italy, Genespire was founded in March 2020 by gene therapy pioneers Prof. Luigi Naldini and Dr. Alessio Cantore, Fondazione Telethon and Ospedale San Raffaele. Genespire is a spin-out of SR-Tiget, a world leading cell and gene therapy research institute. Find out more about us at www.genespire.com. Attachment 240912 Genespire CEO Appointment FINAL

None  Welcome to this week's Chutes & Ladders, our roundup of hirings, firings and retirings throughout the industry. Please send the good word—or the bad—from your shop to Max Bayer or Gabrielle Masson, and we will feature it here at the end of each week.  Lilly’s US leader takes the reins of critical diabetes and obesity unit Eli Lilly With Eli Lilly on the precipice of one of its most consequential FDA approvals ever, the company is making major executive moves.   The company’s head of obesity and diabetes, Mike Mason, is retiring, according to an announcement, just months before the Indianapolis pharma is expected to hear word from the FDA on diabetes med Mounjaro. It's expected to join the likes of Novo Nordisk’s Wegovy as a treatment for obesity. Replacing Mason is Patrik Jonsson, president of the company's U.S. region, who will keep both titles when assumes the new position Jan. 1.  He will relinquish his duties leading Lilly’s immunology unit, however. The company’s chief scientific officer and head of Lilly Research Laboratories, Daniel Skovronsky, M.D., Ph.D., will take over. Skovronsky will be aided by new hire Mark Genovese, M.D., tapped as the senior vice president of immunology. He was the former senior VP of inflammation development at Gilead Sciences and also a professor at Stanford University.  Lilly’s oncology chief David Hyman, M.D., is also getting a title bump, rising to chief medical officer while keeping his cancer oversight duties. To round out the executive rearrangement, Jennifer Oleksiw is being promoted to group vice president and global chief customer officer, and the company's EVP of corporate affairs and communications, Leigh Ann Pusey, is leaving for a new opportunity in the nation’s capital. Fierce Pharma  Deja vu: Flagship plucks another Big Pharma R&D leader Flagship Pioneering  Tell me if you’ve heard this story before: A big-time pharma R&D leader heads for the exits, only to wind up as a top partner at venture creation firm Flagship Pioneering. Well, that tale as old as time (read: the last few years) has added a new chapter. Flagship is adding ex-GSK research leader John Lepore, M.D., to its ranks as CEO-partner, shortly after his departure was announced from the British Big Pharma. He’ll start by taking over ProFound Therapeutics, which last week announced that Klaus Urbahns, Ph.D., would join as CSO. Urbahns was the site head for EMD Serono’s R&D site in Billerica, Massachusetts, which had layoffs earlier this year.  Lepore’s arrival is the third in a recent line of BIg Pharma research leaders to jump to Flagship. Former Bristol Myers Squibb R&D chief Rupert Vessey became Flagship’s chief scientist earlier this year, and Michael Severino jumped from AbbVie last year to lead gene editing biotech Tessera. Fierce Biotech  Gilead’s Kite names new global research chief Kite Pharma New Kite CEO Cindy Perettie has made one of her largest hires since taking the reins, naming Priti Hegde, Ph.D., as the global head of research. The two are reuniting after both working at Foundation Medicine.  Hegde was previously the chief scientific officer at Foundation, steering early research and development of the biotech's genomic profiling work. She’ll now be subbing diagnostics for cell therapy, with Kite looking to maintain its footprint among an ever-growing list of competitors looking to crack CAR-T development.  The company is bolstered by FDA-approved therapies Yescarta and Tecartus, which Kite is working on growing. Yescarta is currently in development as a second-line treatment for high-risk follicular lymphoma, a first-line treatment for high-risk large B-cell lymphoma and a second-line treatment for large B-cell lymphoma in the outpatient setting. Tecartus is being tested as a treatment for pediatric acute lymphocytic leukemia and rare B-cell malignancies. Kite-772 is also in development to treat relapsed and refractory multiple myeloma. Perettie, who joined Kite at the end of May, was the CEO at Foundation while Hegde was CSO. Release > Shephard Mpofu, M.D., has left Novartis after 20 years for immunology-focused Acelyrin, where he will take on the role as SVP of development. Mpofu most recently served as Novartis’ chief medical officer for its gene therapy franchise. Release > Artbio has appointed Nick Pullen, Ph.D., to be chief scientific officer. Before Artbio, Pullen was Jnana Therapeutics' SVP and head of research, and also previously held leadership positions at Celgene/BMS and Pfizer. Release > Jeremy Sokolove, M.D., has left GSK for Odyssey Therapeutics, where he will serve as chief medical officer. Before joining the immuno-oncology biotech, Sokolove was GSK’s SVP and head of clinical pharmacology and experimental medicine; he also previously served as AbbVie’s head of immunology translational science. Release > UniQure is losing a fifth of its employees, including Chief Scientific Officer Ricardo Dolmetsch, Ph.D., who will be retained as a scientific consultant until the end of the year. Richard Porter, Ph.D., will take his place with the combined role of chief business and scientific officer. Fierce Biotech > Swiss biotech Asceneuron has tapped Barbara Angehrn Pavik for the top spot as Dirk Beher, Ph.D., co-founder, original chief scientific officer and CEO since 2014, transitions to the role of chief scientific advisor. Angehrn Pavik has held leadership roles at Amgen, Onyx, Exelixis, CSL's Vifor and was the founder and CEO of Stepstone Pharma. Release > Aadi Bioscience has appointed Dave Lennon, Ph.D., to lead the company, taking the reins from Interim CEO Scott Giacobello, who will continue in his role as chief financial officer.  Lennon joins the oncology biotech from Satellite Bio, where he served as CEO, and has 15 years of experience at Novartis under his belt. Release > Kezar Life Sciences is undergoing a 41% workforce reduction, sending Chief Medical Officer Noreen Henig, M.D., and CEO John Fowler out the door as well. Christopher Kirk, Ph.D., a co-founder of Kezar who served as president and chief scientific officer until this April, will take on the CEO role, while Zung To, senior vice president of clinical development operations, will assume responsibility for clinical trials and drug development operations. Fierce Biotech > Amid a layoff round, Syros Pharmaceuticals’ CEO Nancy Simonian, M.D., has announced her upcoming retirement. Conley Chee, who currently serves as chief commercial officer and chief business officer, will succeed Simonian starting Dec. 2. Fierce Biotech > Broken String Biosciences has looped in two new leaders: Vincent Smith, Ph.D., as chief technology officer and Jessica Rich as VP of business. Before joining the genomics company, Smith held executive-level roles at Base Genomics and Exact Sciences Innovation, while Rich has senior leadership experience from Bionano, Synthego and 54gene. Release  > Aura Biosciences is bringing on Jill Hopkins, M.D., to serve as chief medical officer and president of R&D—replacing former CMO Cadmus Rich, M.D.—and Mark Plavsic, Ph.D., to serve as chief technology officer. Hopkins joins after serving as Novartis’ SVP and global head of ophthalmology and exploratory development, as well as CEO of Novartis’ Gyroscope Therapeutics, while Plavsic joins from Fate Therapeutics, with past experience at Lysogene, Sanofi Genzyme, AstraZeneca, Q-One Biotech and Life Technologies. Release > Twist Bioscience has tapped Mark Buck to serve as SVP of operations. Before joining Twist, Buck was the corporate SVP of operations and global supply chain at Excelitas Technologies Corporation. Release > U.K. biotech Myricx has chosen Francesca Zammarchi, Ph.D., to serve as chief scientific officer. Zammarchi joins from ADC Therapeutics, where she spent 10 years, most recently as head of preclinical pharmacology. Release > Araris Biotech has selected Dragan Grabulovski, Ph.D., to lead the company as acting CEO while founding CEO Philipp Spycher, Ph.D., transitions to the chief scientific officer spot. Grabulovski is an adviser for his practice Grabulovski Consulting Services and the former CSO and co-founder of Covagen, which was acquired by Johnson & Johnson’s Cilag GmbH International in 2014. Release > Alpine Immune Sciences has appointed Wolfgang Dummer, M.D., Ph.D., to serve as chief medical officer, succeeding Andrew Sandler, M.D. Dummer joins from Rigel Pharmaceuticals, where he was CMO, and has past leadership experience at BioMarin and Roche’s Genentech. Release > Nuvig Therapeutics is welcoming Alan Glicklich, M.D., aboard as chief medical officer. Glicklich just departed Chinook Therapeutics, where he was also CMO, and has held past roles across Bird Rock Bio, Arena Pharmaceuticals, Bristol Myers Squibb, Sanofi-Aventis and Regeneron. Release