Angelini Pharma, Inc.

Aptadir Therapeutics is based in Milan Italian biotech Aptadir Therapeutics has launched with the promise that its pipeline of preclinical RNA inhibitors could crack intractable cancers.The Milan-based company was founded by RNA leaders Annalisa Di Ruscio, M.D., Ph.D., of Harvard Medical School's Beth Israel Deaconess Medical Center and Vittorio De Franciscis, Ph.D., of the Italian Research National Council alongside leukemia expert Daniel Tenen, M.D., of the Cancer Science Institute of Singapore and oligonucleotide-focused Marcin Kortylewski, Ph.D., of the City of Hope National Medical Center.At the center of this joint venture is a new class of RNA inhibitors called DNMTs interacting RNAs (DiRs), which are able to block aberrant DNA methylation at a single gene level. The theory is that this reactivates previously hypermethylated genes, considered to be a key feature in cancers as well as genetic disorders.Reactivating specific genes offers the hope of reversing cancers and genetic conditions for which there are either no or limited curative options, such as the blood cancer myelodysplastic syndrome (MDS) in adults and the neurodevelopmental disorder fragile X syndrome in children.Aptadir is hoping to get the most advanced of its DiRs, a MDS-focused candidate dubbed Ce-49, into clinical trials by the end of 2025. To help reach this milestone, the biotech has received $1.6 million in pre-seed funding from the Italian National Technology Transfer Hub’s EXTEND initiative. The hub was set up Italian VC manager CDP Venture Capital SGR. Aptadir is the first biotech to come out the EXTEND initiative, which is partly funded by Rome-based VC firm Angelini Ventures as well as German biotech Evotec.EXTEND’s goal is to “develop high quality science coming from top Italian universities and to help build new start-ups that can develop that science for the benefit of future patients,” CDP Venture Capital’s Claudia Pingue explained in the release.Giovanni Amabile, entrepreneur in residence of EXTEND, has been appointed CEO of Aptadir, having previously helmed autoimmune biotech Enthera.“Aptadir’s business is based on real innovation—a landmark discovery of a new class of molecules which have the potential to be best-in-class therapeutics for intractable conditions,” Amabile said in a Sept. 24 release.“From data already generated, DiRs are highly selective, stable and non-toxic, and have the potential to be used across multiple indications,” Amabile added. “This is a really exciting new field and we are looking forward to pushing our first candidate forward into the clinic.”

MILAN--( BUSINESS WIRE )--Aptadir Therapeutics (Aptadir), a biotech company developing RNA inhibitor-based therapeutics for treating intractable cancers and genetic conditions, announces its launch and the appointment of Giovanni Amabile as Executive Chairman and acting CEO. Aptadir was founded by Dr Annalisa Di Ruscio and Dr Vittorio De Franciscis, both leaders in the field of RNA biology and DNA methylation, alongside Prof. Daniel Tenen, an expert in MDS and leukaemia, and Prof. Marcin Kortylewski, an expert in oligonucleotide immunotherapeutics. They are based at world class institutions: the Beth Israel Deaconess Medical Center of the Harvard Medical School, the Italian Research National Council, the Cancer Science Institute of Singapore and City of Hope National Medical Center. The basis for the Company was the landmark discovery of a new class of RNA inhibitors called DNMTs Interacting RNAs (DiRs) 1,2 . DiRs are capable of blocking aberrant DNA methylation at a single gene level reactivating previously hypermethylated genes – a key feature in cancer and genetic disorders. By reactivating specific genes, there is the opportunity to target the underlying cause of and potentially reverse specific intractable cancers and genetic conditions in adults, such as Myelodysplastic Syndrome (MDS) and, in children, such as Fragile X Syndrome. Aptadir’s proprietary platform can generate multiple DiRs for specific genes suppressed in cancer and genetic conditions, re-establishing the pathway functionality. The first programme, Aptadir Ce-49 is being developed in MDS and the aim is to enter the clinic by the end of 2025. Leading the management team is Giovanni Amabile who has been appointed Executive Chairman and acting CEO; he is also Entrepreneur in Residence of EXTEND. Previously, he was CEO at Enthera and before then, CSO at ADIENNE Pharma. He has a successful track record in raising seed and Series A funds, and for leading IND approvals, complex clinical trials and regulatory approvals in the US. Giovanni Amabile, Executive Chairman and CEO , said: “ Today represents the launch of Aptadir Therapeutics and is an exciting day for the founders and team. Aptadir’s business is based on real innovation - a landmark discovery of a new class of molecules which have the potential to be best-in-class therapeutics for intractable conditions. From data already generated, DiRs are highly selective, stable and non-toxic, and have the potential to be used across multiple indications. This is a really exciting new field and we are looking forward to pushing our first candidate forward into the clinic.” Aptadir has received $1.6m in pre-seed funding from the EXTEND initiative to optimize its lead asset, Aptadir Ce-49. The Company is the first to receive funding from EXTEND, which is the Italian National Technology Transfer Hub wholly dedicated to the biopharmaceutical sector for the development of new therapeutic approaches. The Hub was initiated by CDP Venture Capital SGR, and joint funded with co-investors, Angelini Ventures and Evotec SE. Its purpose is to develop translational drug discovery partnerships with highly renowned universities and research centers in Italy to accelerate their promising therapeutics for commercialization. Claudia Pingue, senior partner and head of the Technology Transfer Fund of CDP Venture Capital SGR , added: “ Aptadir is the first biotech company born from EXTEND, National Technology Transfer hub established on the initiative of CDP Venture Capital and funded with co-investment partners, Angelini Ventures and Evotec. EXTEND aims to develop high quality science coming from top Italian universities and to help build new start-ups that can develop that science for the benefit of future patients. Aptadir represents one of the best examples of where science innovation can create a new potential therapeutic for underserved conditions in adults and children, such as MDS and Fragile X Syndrome. We’ll be watching its progress with great interest.” Di Ruscio A et al 2013, Nature Esposito CL et al 2023, Nature Communication Notes to Editors About Aptadir Therapeutics Aptadir Therapeutics is a preclinical biotech company developing a new class of RNA therapeutics. The Company’s therapeutic focus is underserved intractable cancers and genetic conditions, including underserved intractable conditions in the oncology and rare diseases fields such as Myelodysplastic Syndrome and Fragile X Syndrome. The technology was developed and validated by a group of key opinion leaders in the field of RNA therapeutics based at three world class institutions: the Harvard Medical School, Cancer Science institute of Singapore, Italian CNR Institute and City of Hope National Medical Center. The platform is based on a landmark discovery of a new class of RNA inhibitors called DNMTs Interacting RNAs (DiRs) 1,2 . DiRs are capable of blocking aberrant DNA methylation at a single gene level reactivating previously hypermethylated genes – a key feature in cancer and genetic disorders. The first programme is developing a treatment for Myelodysplastic Neoplasms (MDS), a group of blood cancers that occur when immature blood cells in the bone marrow don't mature into healthy blood cells. Sufferers of MDS go on to develop acute myeloid leukaemia (AML). To find out more, please see: www.aptadir.com About EXTEND EXTEND is the Italian National Technology Transfer Hub (the Hub) wholly dedicated to the biopharmaceutical sector for the development of new therapeutic approaches and it was formed as an initiative of CDP Venture Capital to transform the results of this scientific research into new companies. Alongside CDP Venture Capital, the Hub is funded by co-investors, Evotec, which also acts as an entrepreneurial partner by supporting research groups along the path of scientific study up to the establishment of startups ready to raise capital on the market to start the clinical phase, and Angelini Ventures, the new Angelini Industries Group company operating in the VC sector with a focus on digital health and biotech. The scientific promoters of the initiative are IRSCC San Raffaele Hospital in Milan, the Universities of Milan, Florence, Modena and Reggio Emilia, Padua, Trento, La Sapienza in Rome, the regional research district of Puglia through H-Bio, and Human Technopole. To learn more, please visit: www.extend-tt.vc

With a career dedicated to delivering firsts in medicine and innovating to find new ways to treat patients, McKerracher’s expertise will help Genialis commercialize first-in-class biomarkers. BOSTON--(BUSINESS WIRE)-- Genialis, the RNA-biomarker company, today announced the appointment of Krista McKerracher as Chairperson of the Board of Directors. McKerracher is a seasoned biopharmaceutical leader passionate about bringing new medicines to patients with life-threatening diseases. Her background includes almost three decades at Johnson & Johnson and Novartis Oncology, where she held senior commercial and senior R&D leadership roles and launched several first-in-class drugs in the hematology/oncology space. McKerracher rounded out her drug development career at CRISPR Therapeutics, where she led the team that brought the first CRISPR Cas9 ex-vivo gene editing product to the clinic (HbF edited stem cells for hemoglobinopathies.) This press release features multimedia. View the full release here: Genialis Introduces Krista McKerracher as Chairperson of the Board. Pictured from left to right is the Genialis Board of Directors: Franck Brichory, Debiopharm Innovation Fund (observer); Cédric Odje, Debiopharm Innovation Fund; Krista McKerracher, Fig Advisory; Rafael Rosengarten, PhD, CEO & co-founder, Genialis; Miha Štajdohar, PhD, CTO & co-founder, Genialis; Elia Stupka, PhD, Angelini Ventures; Ita Lu, Taiwania Capital; Paoyen Lai, Taiwania Capital (observer) (Photo: Business Wire) McKerracher joins Cedric Odje, representing Debiopharm Innovation Fund; Ita Lu of Taiwania Capital; independent director Elia Stupka; and Genialis co-founders Rafael Rosengarten, Ph.D. (chief executive officer) and Miha Štajdohar, Ph.D. (chief technology officer) on the board. “Throughout my career, I’ve worked on a lot of firsts. Genialis has a promising opportunity to be the first to harness the vast amount of data contained in a small tumor sample and use that to bring reality to truly personalized medicine – delivering the right drug for the right patient at the right time,” said McKerracher. “When I was diagnosed with cancer in 2015, tumor sequencing was still brand new, and despite working in the field, many of my colleagues were skeptical about the value of sequencing for my tumor type because links to specific mutations were not well understood. With the Genialis approach to RNA-based biomarkers, I believe that in the future, all patients will have their tumors sequenced as standard of care. Genialis is on the leading edge of precision medicine, driving innovation and hope for patients globally.” Ms. McKerracher is an experienced public company board member known for bringing a solid commercial orientation to the drug development process. McKerracher graduated from the University of Waterloo (BSc. Hon. Applied Health Sciences) and earned an MBA from York University. “When we started our search for a board chair, we focused on finding someone who embraced our company value of ‘people first’ and committed to improving outcomes for patients globally,” said Dr. Rosengarten. “Krista brings the perspective of someone who has overseen the entire drug development life cycle on a global scale for some of the preeminent pharma companies, helping Genialis direct its biomarker algorithms to the most critical pain points in the process.” Genialis recently announced a breakthrough development in its predictive biomarker for KRAS inhibitor therapies and is currently enrolling select participants in its early access program. Genialis krasID is a new Artificial Intelligence classifier that accurately predicts response and clinical benefit to KRAS inhibitors across preclinical, clinical, and real-world settings. Most biomarkers in the KRAS space measure only KRAS mutation status, which may be necessary to receive the drug but is insufficient to predict or monitor response or to inform combinations or subsequent therapeutic interventions. By using high-dimensional gene expression data, Genialis krasID captures underlying biological complexity unique to each individual patient. Over 50 biopharma companies are currently developing more than 70 different KRAS-targeted drugs across over 12 disease indications. The total KRAS therapy market today is estimated at approximately $240 million, with a compound annual growth rate of 36% to reach $10 billion by 2032. Meanwhile, available diagnostics to support KRAS-targeted patient care are limited to mutation-based tests. Genialis is participating in the Festival of Genomics & BioData this week in Boston. Dr. Rosengarten will give a talk in the Live Lounge titled, “Genialis™ krasID: Biology-Driven Machine Learning to Personalize KRAS Inhibitor Therapy” on Wednesday, June 12, 2024, from 12:10 PM to 12:40 PM EDT. To schedule a meeting at the Frontline Genomcis event, or learn more about Genialis biomarker programs, please visit or email biomarkers@genialis.com About Genialis Genialis, the RNA biomarker company, is creating a world where healthcare delivers the best possible outcomes for patients, their families, and their communities. Genialis develops and validates clinically actionable biomarkers informed by the world’s most ethnographically diverse cancer data sets to better predict patient responses and guide treatment decisions for targeted inhibitors, immunotherapies, and other emerging therapeutic classes. Genialis is trusted by pharma and diagnostics partners, and together, we are transforming medicine through data. For more information, please visit . View source version on businesswire.com: Contacts Source: Genialis Smart Multimedia Gallery Photo Genialis Introduces Krista McKerracher as Chairperson of the Board. Pictured from left to right is the Genialis Board of Directors: Franck Brichory, Debiopharm Innovation Fund (observer); Cédric Odje, Debiopharm Innovation Fund; Krista McKerracher, Fig Advisory; Rafael Rosengarten, PhD, CEO & co-founder, Genialis; Miha Štajdohar, PhD, CTO & co-founder, Genialis; Elia Stupka, PhD, Angelini Ventures; Ita Lu, Taiwania Capital; Paoyen Lai, Taiwania Capital (observer) (Photo: Business Wire) Logo View this news release and multimedia online at: