Day 2 – Thursday 6th March 2025 - CET (Central European Time, GMT+01:00)
The RNA Leaders ‘Women in RNA (WiRNA) Forum’ is a dedicated space to empower and connect women and allies who are shaping the future of RNA research and therapeutics.
Networking Breakfast to hear and learn about the career journeys of women leaders in the space.
- Shalini Andersson - Vice President of Oligonucleotide Discovery, AstraZeneca
- Malgorzata Gonciarz - Global Head xRNA Therapeutics, Novartis
- Aurélie Goyenvalle - Director, INSERM
- Tamar Grossman - VP, Global Head of RNA, Gene Therapy and Delivery, Johnson & Johnson Innovative Medicine
- Jeff Milton - CEO, La Holla Labs
- Potential of oligo therapeutics in CNS/neurological indications
- Manufacturing and green chemistry processes for oligos
- Immunogenicity of oligonucleotide-based drugs
- Delivery and conjugates for small RNAs
- Incorporating AI in oligo drug discovery and development
- Stefan McDonough - Senior Vice President, Head of Neuroscience, Atalanta Therapeutics
- Sonja Merkas, Ph.D. - Founder, Livinovea
- Shalini Andersson - Vice President of Oligonucleotide Discovery, AstraZeneca
- Jeff Milton - CEO, La Holla Labs
- Alan Whitmore - CSO, e-therapeutics
- Developing a personalised mRNA vaccine & therapeutics – CMC challenges and considerations
- Innovating mRNA therapeutic dosing and administration
- Clinical progression of mRNA therapeutics & vaccines
- Advancing Next-Generation Delivery Solutions for mRNA
- Optimizing mRNA targeting and characterisation
- Steve Pascolo - Professor, University Hospital of Zurich
- Simone Carneiro - Postdoctoral Fellow, LMU Munich
- Tamar Grossman - VP, Global Head of RNA, Gene Therapy and Delivery, Johnson & Johnson Innovative Medicine
- Arun Kumar - RNA Innovations Programs Leader, CEPI
- Generoso Ianniciello - CBO, Anima Biotech
- Beatriz Llamusi - CSO, Arthex Biotech
Positive topline 48-week data from the ALPACAR-360 Phase 2 study of zerlasiran in patients with elevated lipoprotein(a) (Lp(a)) with highly significant and sustained reductions in Lp(a) to week 48 (end of treatment period)
Zerlasiran was well tolerated
Data support advancing zerlasiran into Phase 3
- Marie Wikstrom-Lindholm - CSO, Silence Therapeutics
- Marketed oligonucleotide drugs
- Modification of oligonucleotides and their advantages
- Development strategies to control related substances
- Asymchem oligonucleotide CDMO service capability and capacity
- Balasubramanian Arumugam - Director, Asymchem
- Accurate prediction of drug properties, such as activity, stability, and off-targets will lead to faster, cheaper drug development and fewer animal experiments.
- Abzu specializes in in silico drug design, using explainable AI as the foundation of our approach, with continuous development of new models to predict diverse drug properties.
- Our latest work focuses on predicting off-target effects from RNA sequencing data, with significant attention to data processing, feature generation, and advanced modeling techniques.
- The siRNA candidates selected using our approach differ significantly from traditional off-target filtering methods, offering a more effective and tailored drug design strategy.
- Disa Tehler - Head of RNA Therapeutics, Abzu
DM1 is a rare neuromuscular disease with no disease modifying treatments. Arthex’s therapeutic approach in DM1 is miR-23b inhibition, which demonstrated a dual beneficial effect on DM1 models
In order to alleviate functional and molecular symptoms of DM1 disease, preferentially in the tissues affected by the disease, Arthex is developing a safe lipid-conjugated antimiR-23b oligonucleotide (ATX-01)
Explore how the conjugated antimiR reaches muscle, heart, brain and diaphragm more efficiently than the naked molecule in the HSALR mice and shows improved PK/PD properties. Importantly, the level of compound delivered into brain by IV injection in the DMSXL animals was enough to produce important target engagement and concomitant rescue of exploratory behaviour of the mice
- Beatriz Llamusi - CSO, Arthex Biotech
- Ariane Gomes - CSO, baseimmune
- Optimizing self-replicating RNA and manufacturing drive and increase in immunogenicity and safety for a best-in-class therapeutic index
- Optimized self-replicating RNA have durability that matches conventional vaccine technologies
- An improved therapeutic index drives advantages in vaccines targeting multiple antigens in complex pathogens or combination vaccines
- Nathaniel Wang - CEO, Replicate Bioscience
- Reviewing factors in manufacturing site design for sustainable and rapid production of both mRNA vaccines and therapeutics
- Exploring the needs of mRNA vaccine and therapeutic manufacturing processes to be fully continuous, and exploring the latest solutions
- Harris Makatsoris - Academics Professor of Sustainable Manufacturing Systems, King's College London
Advancing mRNA-based ATAK CAR therapy to detect and eliminate GPC3-advanced hepatocellular carcinoma, with an update on phase 1 patient dosing
Discussing clinical progression of secondary program to treat GPC3-breast cancer
- Daniel Getts - CEO, Myeloid Therapeutics
- Lorna Harries - CSO, Senisca
Creating an AI-designed endless RNA (eRNA) and programable natural nanoparticles, to achieve precise delivery and extended therapeutic durability in patients with cancer, metabolic disease, infectious disease, rare disease and autoimmune disorders
Insight into preclinical data and movement into the clinic
- Trevor Hallam - CSO, Sail Biomedicines
- Divalent siRNA (di-siRNA), a novel small interfering RNA technology, has potency, CNS distribution, and durability appropriate for neurological disease
- ATL-201, a di-siRNA targeting the KCNT1 sodium-activated potassium ion channel, gives selective knockdown of KCNT1 transcript and protein and reaches most neurons in the cortex following a dose into cerebrospinal fluid
- ATL-201 gives dose-dependent reduction in seizures lasting over four months in mice with Kcnt1-driven epilepsy and shows promise for treatment of gain-of-function KCNT1 genetic epilepsy
- Stefan McDonough - Senior Vice President, Head of Neuroscience, Atalanta Therapeutics
- Elevating endogenous utrophin protein expression via the RNAa mechanism
- Delivering duplex RNA to muscle and heart by proprietary LiCO platform
- in vivo evaluation of PD and efficacy in utrophin humanized mdx mice
- Long-Cheng Li - CEO, Ractigen Therapeutics
- Mikko Turunen - CSO, RNatives Inc.
- KRRO-110 is the first RNA editing oligonucleotide product candidate from Korro’s proprietary RNA editing platform, Oligonucleotide Promoted Editing of RNA (OPERA™). KRRO-110 is designed to co-opt an endogenous enzyme, Adenosine Deaminase Acting on RNA’s (ADAR), to edit the “A” variant on SERPINA1 RNA, repair an amino acid codon, and restore secretion of normal AAT protein.
- This repair of the endogenous protein has the potential to clear protein aggregates from within liver cells to create a potentially clinically differentiated benefit for liver function and to preserve lung function by providing an adequate amount of normal AAT protein.
- Kemi Olugemo - CMO, Korro Bio
- John Taylor - Global Head of Business Development, Ribocure
- Progressing a novel circRNA approach to improve patient outcomes with Hantaan virus and Yellow Fever virus
- Crafting an innovative nanoparticle delivery technology that ensures room temperature stability
- Peter Weinstein - CEO, EmerVax
- Design and development of anti miR-22 ASO
- Preclinical evidence in obesity
- Combination with available therapies to improve clinical outcome
- Riccardo Panella - CSO, Resalis Therapeutics
Discussing the role of small interfering RNA (siRNA) technology in targeting lipoprotein(a) (Lp(a)), a risk factor for cardiovascular disease
Explaining how siRNA-based therapies can be used to assess cardiovascular risk by measuring Lp(a) levels and evaluating the potential benefits of Lp(a) lowering
Exploring the potential clinical applications of siRNA-based Lp(a) lowering therapies, including their use in high-risk patients and their integration into current cardiovascular risk management strategies
- Christopher O’Donnell - Global Head Cardiovascular and Metabolism Translational Medicine, Novartis BioMedical Research
Discuss the results of the Phase 3 study demonstrating the efficacy of vutrisiran in treating cardiomyopathy, including improvements in cardiac biomarkers and patient outcomes
Discuss the future development plans for vutrisiran, such as exploring additional indications or combination therapies, and the potential impact on the treatment of cardiomyopathy
- Paul Nioi - SVP, Alnylam Pharmaceuticals
Arrowhead is developing ARO-INHBE, an RNAi-based therapeutic designed to reduce the production of INHBE, a protein involved in regulation of adipose storage
Another RNAi-based therapeutic, ARO-ALK7, is being investigated to target ALK7, an adipocyte receptor implicated in obesity and metabolic disorders
Clinical Trials - Arrowhead plans to initiate clinical trials for both ARO-INHBE and ARO-ALK7 in early 2025, aiming to evaluate their safety and efficacy in treating obesity
- James Hamilton - Chief of Discovery & Translational Medicine, Arrowhead Pharmaceuticals
Delving into the LUNAR Delivery Platform
Discussing clinical progression of ARCT-810 and ARCT-032 programs to improve outcomes for patients with Cystic Fibrosis and Ornithine Transcarbamylase (OTC) Deficiency
- Pad Chivukula - CSO, Arcturus Therapeutics
- Updates on Phase 1 & 2 clinical candidates against influenza, RSV and acne vulgaris, and sharing outlook on future indications
- Discussing how integrated translational science enables us to improve immunogenicity and reactogenicity of mRNA vaccines
- Jean-François Toussaint - Global Head of R&D Vaccines, Sanofi
- MGMT-unmethylated glioblastoma has a poor prognosis, with a median overall survival of approximately 12 months after surgery and chemoradiation with temozolomide
- mRNA vaccines have been shown to induce CD4+ and CD8+ T cell responses against a variety of cancer antigens and offer the possibility to encode multiple antigens on a single construct
- Results from an ongoing phase 1 clinical trial evaluating the safety and immunogenicity of CVGBM, an investigational multiantigen mRNA vaccine, in patients with newly-diagnosed and surgically resected MGMT-unmethylated glioblastoma show promising immunogenicity with good tolerability
- Myriam Mendila - Chief Scientific Officer, CureVac
The RNA Leaders CXO Forum provides a unique, powerful and valuable space for candidly sharing ideas and experiences between executives of ~20 RNA Biotech private companies.
This forum will include 2-3 case studies discussing the right approach to engage and partner with pharma and industry assessing the key factors why pulling together a biotech valuation, from an investor, pharma and biotech perspective. It will also include a general discussion focused on brainstorming solutions to the collective challenges of CEO/CSO/CBOs in the sector.
Highlighting EUpALS' role in advocating for patients' needs and driving progress in ALS research and treatment development
Discussing the current state of patient-physician-researcher dynamics and identify areas where collaboration can be enhanced
Exploring patient perspectives on adopting emerging therapies, such as RNA/nucleic acid-based treatments, and discussing strategies for facilitating their uptake
Sharing insights into EUpALS' involvement in ALS trials utilizing RNA/nucleic acid-based therapies, such as Wave LifeSciences and Biogen
- Evy Reviers - Chairwoman, EUpALS
What technologies and scientific platforms are considered as next-gen RNA?
How is the field advancing in RNA biology and technology platforms to be ready for next-gen applications?
What top approaches and indications are being fast-tracked to the clinic for both small and long RNAs?
- Paul Nioi - SVP, Alnylam Pharmaceuticals
- Myriam Mendila - Chief Scientific Officer, CureVac
- Hendrik Knoetgen - Global Head of Nucleic Acid Based Research, Roche
- Michelle Lynn-Hall - Associate Vice President of Genetic Medicine, Eli Lilly
- Sonja Merkas, Ph.D. - Founder, Livinovea
- Renee Williams - Founder and Managing Partner, Williams Biotech Consulting