Presenters of 24th BEF

20-MINUTE PRESENTERS:

BiotechnologyPharmaceuticals/Licensing

CNS Pharmaceuticals, Inc.

Houston, United States

CNS Pharmaceuticals (NASDAQ:CNSP) is a clinical-stage pharmaceutical company developing a pipeline of anti-cancer drug candidates for the treatment of primary and metastatic cancers of the brain and central nervous system.

Website

Biotechnology

Herantis Pharma Plc

Espoo, Finland

Wed, 25 Sep · 14:15 – 10:30

Herantis Pharma Plc is a clinical-stage biotechnology company developing disease modifying therapies for Parkinson’s disease. Herantis’ lead product HER-096, is an advanced small synthetic chemical peptidomimetic molecule developed based on the active site of the CDNF protein. It combines the compelling mechanism of action of CDNF with the convenience of subcutaneous administration. The Phase 1a clinical trial demonstrated a good safety and tolerability profile, and efficient blood-brain barrier penetration of subcutaneously administered HER-096 in humans. The shares of Herantis are listed on the Nasdaq First North Growth Market Finland.

Website

Biotechnology

IAMA Therapeutics S.r.l.

Genoa , Italy

Wed, 25 Sep · 14:35 – 10:30

IAMA Therapeutics is a clinical-stage biopharmaceutical company committed to developing innovative treatments for central nervous system disorders. The lead compound, IAMA-6, is a selective NKCC1 inhibitor designed to restore chloride homeostasis and rebalance GABAergic signaling. The mechanism has been studied in multiple CNS disorders, including Neurodevelopmental Disorders, such as Fragile X, Down Syndrome and Autism Spectrum disorders, and psychiatric and neurological conditions.

With a successfully completed phase 1 study, IAMA-6 is progressing to a Phase 2a proof of concept study in Fragile X which will be initiated late 2026.

IAMA is funded through phase 2 and is seeking funds to expand the clinical development program.

Website

Biotechnology

MetrioPharm AG

Zurich, Germany

Wed, 25 Sep · 13:35 – 10:30

MetrioPharm is a Swiss clinical-stage biotech company that has developed a platform of oral small molecule immune modulators that normalize the pathologically dysregulated (reprogrammed) mitochondrial energy metabolism in macrophages and other immune cells without being immunosuppressive. This plays an important role in most inflammatory and infectious diseases. MetrioPharm's lead candidate MP1032 has a unique, self-regulating mechanism of action that makes it a first-in-class ROS (Reactive Oxygen Species) scavenger that reduces excessive intracellular ROS (intracellular oxidative stress) WITHOUT (!) interfering with the physiological (normal) ROS levels of other cells. ROS are essential for cell signaling. MP1032 has demonstrated broad-spectrum anti-inflammatory and antiinfectious activity in three completed Phase IIa clinical proof-of-concept studies in Psoriasis and COVID-19 involving a total of 234 patients treated with MP1032. In addition, MP1032 has shown strong efficacy data in preclinical in vivo studies in other inflammatory diseases such as Rheumatoid Arthritis, Multiple Sclerosis, Inflammatory Bowel Diseases, Sepsis and, most recently, Duchenne Muscular Dystrophy. As a monotherapy, MP1032 has therapeutic effects similar to corticosteroids, but without serious side effects. MP1032 in combination with ultra-low dose corticosteroids has the potential to replace the current standard of care, high dose corticosteroid therapy, with increased efficacy (2.5 times the high dose) and significantly reduced side effects. MP1032 has also demonstrated a broad host-directed antiviral and antibacterial activity in preclinical and clinical studies, which would be essential for improving future pandemic preparedness. MetrioPharm is initially focusing its anti-inflammatory drug development on rare diseases such as Duchenne Muscular Dystrophy. In this indication, patients are treated for many years or even decades with high-dose corticosteroids (cortisone-based therapeutics) as standard anti-inflammatory therapy, which typically leads to severe side effects. The focus on orphan indications is the fastest route to conditional marketing authorization by the EMA and accelerated approval by the FDA for MP1032.

Website

Biotechnology

Nouscom AG

Basel, Switzerland

Wed, 25 Sep · 14:35 – 10:30

Nouscom is a private clinical stage immuno-oncology company developing next-generation, off-the-shelf and personalized cancer immunotherapies. Our proprietary viral vector platform has the capacity to encode for large payloads of neoantigens or other immunomodulators and is clinically demonstrated to safely and potently harness the power of the immune system. The company recently closed an oversubscribed Series 'C' financing, to advance the clinical development of its wholly owned programs. These programs include NOUS-209, an off-the-shelf cancer immunotherapy for the treatment of MSI-H solid tumors in combination with pembrolizumab in randomized phase 2 trials, with readouts expected in 2025. In January 2023, Nouscom announced a clinical trial collaboration and pembrolizumab supply agreement with MSD (Merck & Co). A Phase 1b study of NOUS-209 monotherapy in Lynch Syndrome Carriers with potential to ‘intercept cancer’ before it occurs is running with the National Cancer Institute, with the first interim data reported at SITC Conference in November 2023. A randomized pivotal trial in Lynch Syndrome is expected to be initiated in 2025. Nouscom's second clinical program is NOUS-PEV, a personalized cancer immunotherapy for the treatment of advanced melanoma and lung cancer that will be entering randomized Phase 2 trials in 2025. Nouscom has also exclusively out-licensed VAC-85135, an off-the-shelf immunotherapy developed under a multi-project agreement, which is currently under evaluation in a Phase 1 trial for the treatment of Myeloproliferative Neoplasms sponsored by Janssen Research & Development and Bristol-Myers Squibb.

Website

Oryzon Genomics, S.A.

Spain

Wed, 25 Sep · 15:15 – 10:30

A public clinical-stage biopharma company developing epigenetics for CNS disorders and oncology/hematology. c.50 highly-qualified professionals located in Barcelona, Boston and San Diego. Listed in Spain, aiming at NASDAQ-listing. Two uncorrelated clinical-stage compounds: vafidemstat (Phase III-ready, CNS) and iadademstat (Phase II, oncology/hematology). Pioneering development of epigenetic drugs in CNS with vafidemstat, a safe LSD1 inhibitor, administered to + 425 subjects. The drug has shown promising results in reducing agitation/aggression in psychiatric patients in a Phase IIa basket trial, and in a global randomized, double blind Phase IIb trial in BPD (PORTICO, final results presented at ECNP-2024). Phase III in agitation/aggression in borderline personality disorder (BPD) in preparation. Vafidemstat is also being investigated for treating negative symptoms of schizophrenia in a randomized, double-blind Phase IIb trial (ongoing trial expansion to additional EU countries). Preparing a new Phase II trial in aggression in patients with ASD, including genetically-defined ASD subpopulations such as Phelan McDermid Syndrome. Iadademstat, a best-in-class LSD1 inhibitor, is in clinical development for AML, solid tumors and hematological disorders. PoC with strong clinical activity in combo with azacitidine in a Phase II in unfit AML patients, and encouraging signals in 2L-ED-SCLC. Highly encouraging data presented at ASH-2025 from ongoing trials in 1L AML (in combo with venetoclax/azacitidine, 100% ORR, 90% CR) and in R/RFlt3mut+ AML (in combo with gilteritinib, 67% CCR at the dose under expansion). Additional trials ongoing in MDS (in combo with azacitidine),1L SCLC (in combo with ICI), and in MPN (in combo with ASTX727). Expanding into hematological indications: sickle cell disease (PhIb trial approved by EMA, recruiting), essential thrombocythemia (PhII in prep).

Website

STALICLA SA

Switzerland

Wed, 25 Sep · 14:55 – 10:30

STALICLA SA is a Swiss clinical-stage biopharmaceutical company, revolutionizing the treatment of neurodevelopmental (NDD) and wider neuropsychiatric disorders. The company has developed a clinically validated neuro precision development platform, successfully delivering stratified patient subgroups and advancing tailored treatment options. Its lead NDD asset, STP1, and a second NDD asset are set to enter Phase 2 trials with expected readouts within 30 months. STP7 (Mavoglurant), is also being advanced for Phase 3 trials, fully funded by the US government with discussions underway for out-licensing. We are currently raising a series C of $60m to recite the Phase 2 precision autism program development.

Website

BiotechnologyPharmaceuticals/Licensing

TME Pharma

Berlin, Germany

Wed, 25 Sep · 14:15 – 10:30

TME Pharma is a clinical-stage biotech company developing innovative therapies for aggressive cancers, focusing on the tumor microenvironment (TME). Our lead compound, NOX-A12, targets CXCL12 and has received Fast Track designation from the FDA for newly diagnosed, chemotherapy-resistant glioblastoma. In a Phase 1/2 trial, NOX-A12 combined with anti-VEGF and radiotherapy significantly increased survival compared to the standard of care reference cohort (p=0.003, Hazard Ratio: 0.30) and patients receiving NOX-A12 with radiotherapy alone (p=0.021, Hazard Ratio: 0.34). Median survival improved from 9.5 months to 19.9 months. A randomized, controlled Phase 2 trial is approved in the US and Germany to test multiple doses of NOX-A12 with anti-VEGF and radiotherapy. NOX-E36, our second clinical-stage asset, has demonstrated safety, tolerability, and promising pharmacokinetics/pharmacodynamics (PK/PD) as an anti-fibrotic treatment in ophthalmology. We plan to monetize NOX-E36 through a spinout or similar structure.

Website

10-MINUTE PRESENTERS:

BiotechnologyDrug DeliveryPharmaceuticals/Licensing

Ageronix SA

Geneva, Switzerland

Thu, 26 Sep · 15:50 – 10:30

Ageronix SA is a clinical-stage life sciences company that is developing therapies for diseases of the PNS and CNS. Ageronix's lead program is focused on Charcot-Marie-Tooth Disease Type 1A (CMT 1A). Ageronix has also programs in development for Neuropathic Pain and Alzheimer's Disease.

Website

Biotechnology

Alder Therapeutics AB

Solna, Sweden

Wed, 25 Sep · 12:20 – 10:30

We are one of the leaders in Replacement Cell Therapy and aim to treat Retinitis Pigmentosa, an inherited blindness with almost no treatment options today. We have shown our pre-clinical and CMC to both FDA and EMA and received positive feedback to continue our development. Our manufacturing is similar to biologics both in manufacturing and price, we can produce a dose for less than 10 000 €. We are raising 22 M€ to generate clinical proof of concept in a small Phase I/IIa trial where we expect to get secondary efficacy data. From there it is expected that we only need 50-100 more patients in a Phase III study to apply for market approval. We are building a syndicate now and are looking for the final partner. Will you join us and give hope to the Retinitis Pigmentosa patients?

Website

Biotechnology

Atamyo Therapeutics SAS

Evry-Courcouronnes, France

Wed, 25 Sep · 11:40 – 10:30

Atamyo Therapeutics is a clinical-stage biopharma focused on the development of a new generation of effective and safe gene therapies for muscular dystrophies and cardiomyopathies. A spin-off of gene therapy pioneer Genethon, Atamyo leverages unique expertise in AAV-based gene therapy and muscular dystrophies from the Progressive Muscular Dystrophies Laboratory at Genethon. Atamyo has a clinical-stage pipeline with first-in-class vectors which include new capsids, new promoters, and organs-detargeting technologies: - ATA-100 is in phase 1b/2b in LGMD-R9/LGMD2I associated to deficiencies in the protein FKRP. - ATA-200 has received clinical trial clearance for LGMD-R5 related to deficiencies in γ-sarcoglycans, - ATA-300 is in IND-enabling studies and targets LGMD-R1/ LGMD2A or calpainopathy; - The cardiomyopathy programs pursues several targets in Dilated Cardiomyopathies Atamyo’s seasoned management has a unique expertise in developing biotech products from Research to late stage clinical development: - Stephane Degove, its CEO, is a biotech entrepreneur with 25 years’ experience in pharma/biotech and strategy. - Isabelle Richard, PhD, its Chief Scientific Officer, heads the Muscular Dystrophy department at Genethon and a pioneer in the research on gene therapy targeting muscular distrophies. - Dr Sophie Olivier, its Chief Medical Officer, has extensive clinical development experience in large and small pharma organizations and has overseen multiple regulatory interactions with both the FDA and the EMA, particularly in regard to pediatric development and rare diseases. - Catherine Cancian, its Chief Technical Officer, has 25 years CMC development in biologics, including gene therapy. The name of the company is derived from two words: Celtic Atao which means “Always” or “Forever” and Myo which is the Greek root for muscle. Atamyo conveys the spirit of its commitment to improve the life of patients affected by neuromuscular diseases with life-long efficient treatments.

Website

Biotechnology

Atopia Therapeutics

Geneva, Switzerland

Wed, 25 Sep · 16:30 – 10:30

Atopia Therapeutics is a Geneva-based biotech startup developing a revolutionary biological treatment for type 2 allergic diseases such as asthma, atopic dermatitis, allergic rhinitis and food allergy, based on a natural immunomodulatory protein.

Website

Pharmaceuticals/Licensing

Avata Biosciences Ltd.

London, United Kingdom

Wed, 25 Sep · 10:40 – 10:30

Avata Biosciences is a privately owned, clinical stage pharmaceutical company with lead asset AVAT-021 in development for adult focal-onset seizures (FOS), using the 505(b)(2) regulatory pathway with Epidiolex® as reference product. A Phase 3 study in FOS is planned for 2026. Avata is also in partnership with Oxford University to improve mental health and aims to start a Phase 2 efficacy study with AVAT-021 in patients with Schizophrenia in 2025. AVAT-021 is a solid-dose formulation of cannabidiol (CBD) with highly encouraging PK data from a comparative Phase 1 study against Epidiolex® (CBD dissolved in oil) which completed in 2024. A wealth of clinical data is available to support the efficacy of cannabidiol in both epilepsy and schizophrenia. Recent company achievements: • Optimization of AVAT-021 to achieve Target Product Profile • Improved bioavailability compared to Epidiolex® in a Phase 1 crossover study • Pre-IND meeting with FDA confirming access to 505(b)(2) pathway • Selection of commercial scale manufacturing sites (US & ex-US) Avata Biosciences is currently seeking funding to further progress the AVAT-021 clinical development programs and is in discussion venture capital groups, as well as exploring strategic partnering opportunities with pharmaceutical companies.

Website

Biotechnology

BiPER Therapeutics

Strasbourg, France

Wed, 25 Sep · 12:10 – 10:30

BiPER Therapeutics is developing oral First In Class small molecules against cancer. BPR001-615 our lead clinical candidate, at 9 months from the clinic, pushes cancers cells to burn out - a unique mechanism of action. BPR001-615 pave the way for new therapeutics based on our unique science and technology overstressing pathological cells to cure patients.

Website

Bloom Science, Inc.

San Diego, United States

Wed, 25 Sep · 14:05 – 10:30

Bloom Science is redefining medicine by creating innovative therapies that harness the body’s natural metabolic and neurological pathways. Inspired by the therapeutic benefits of the ketogenic diet, Bloom’s approach targets multiple mechanisms simultaneously to provide safer, more effective solutions for obesity, Dravet syndrome, ALS, and other complex conditions. Our lead program, BL-001, is a live biotherapeutic product reverse engineered to replicate the ketogenic diet’s clinical efficacy. BL-001 has demonstrated proof of mechanism and has been observed to be safe and tolerable, with Bloom planning Phase 2 clinical trials in both obesity and Dravet syndrome. The IrisRx™ discovery platform powers Bloom’s pipeline by integrating cutting-edge science and a proprietary knowledgebase. This platform enables the development of novel, multi-pathway therapeutics through gut commensal microbes and synthetic biology, delivering innovative treatments with broad therapeutic potential. Backed by top-tier investors and a team of industry veterans, Bloom Science is advancing a deep pipeline to transform patient care and create lasting clinical impact.

Website

Biotechnology

CIS BIOPHARMA AG

Bubendorf, Basel, Switzerland

Wed, 25 Sep · 11:30 – 10:30

CIS BIOPHARMA – Pioneering Targeted Cancer Therapies CIS BIOPHARMA helps cancer patients live longer, better lives. Located in the Basel area, Switzerland, we are a biotech company with over 50 years of activity in Life Sciences. We develop the next generation of targeted cancer therapies with a focus on Antibody Drug Conjugates, ADCs, and radiopharmaceuticals. We target cancers with high unmet medical need by addressing both established and emerging oncology targets. Through our proprietary platforms, we create first-in-class and best-in-class immunoconjugates designed to improve patient outcomes. Our technologies inlcude advanced protein engineering, linker and payload design, and bioconjugation, enabling innovative approaches to precision oncology.

Website

DiagnosticsDigital Health

éclateral Ltd.

Bristol, United Kingdom

Wed, 25 Sep · 10:30 – 10:30

éclateral's o~pal platform combines two robust technologies that are already manufactured at scale to deliver a low-cost, clinical grade health and wellbeing testing platform suitable for use in the home or at point of care. Our first test, for C-reactive protein (CRP), is intended to be used to guide the prescription of antibiotics for patients with respiratory tract infections. There is considerable regulatory push and market pull for this test globally. Three tests have been approved for use in the UK and a clinical care pathway is in place, but the competitor tests are too expensive. Our combination of electrochemistry and lateral flow testing means that we have produced a best-in-class semi-quantitative test that is attractive to users in primary care as well as in care home settings. CRP testing is already used to guide antibiotic prescriptions in countries across Europe and our low cost will allow us to compete effectively in these markets. It is also worth noting that CRP is probably the most tested protein in medicine, and that there are opportunities for a fully-quantitative CRP test to guide treatments in chronic diseases settings too, notable in rheumatoid arthritis and Crohn's disease. Our planned second test will initially be marketed at women experiencing symptoms of the perimenopause. At any given time this affects 3 million women in the UK and there is clear demand for a test to explain the distressing symptoms that affect a large proportion of women, 10% of whom have to leave work early when symptoms are very severe. The test will measure 4 sexual health hormones, 4 dietary markers, 3 thyroid markers and cortisol (the "stress hormone") and so will be useful beyond the initial target market for men experiencing general tiredness as well as for women seeking fertility testing.

Website

BiotechnologyPharmaceuticals/Licensing

Eleva GmbH

Freiburg, Germany

Wed, 25 Sep · 16:00 – 10:30

Eleva is a clinical-stage biopharmaceutical company discovering and developing previously inaccessible biological therapeutics. Eleva’s disruptive moss-based technology platform enables GMP-scale manufacturing of human proteins with tremendous therapeutic potential that have been too challenging to manufacture using other approaches. The company’s proprietary pipeline includes candidates for complement disorders and enzyme replacement therapies. The lead program, CPV-104 recombinant human complement Factor H, is in Phase 1b testing to treat C3 Glomerulopathy (C3G). An intravitreal formulation of the candidate is in late preclinical development to treat dry AMD. The company’s aGal (RPV-001) program has completed a positive Phase 1b single-dose clinical trial to treat Fabry disease.

Website

Biotechnology

FoRx Therapeutics AG

Basel, Switzerland

Wed, 25 Sep · 13:55 – 10:30

FoRx Therapeutics is a preclinical-stage company dedicated to the discovery and development of innovative, next-generation oncology drugs that target DNA Damage Response (DDR) pathways in cancer. Our lead program is a potent and selective PARG inhibitor (FORX-428) with strong evidence for best-in-class potential, which was recently declared as Development Candidate and is currently progressing to IND.

Website

Biotechnology

Fusix Biotech GmbH

Munich, Germany

Thu, 26 Sep · 13:45 – 10:30

Fusix Biotech is an early-stage university spinout based in Munich, Germany. They are developing a completely novel, synthetic oncolytic virus platform that offers potent tumor cell killing via a unique mechanism of action, modulatory activity in the tumor microenvironment and induction of systemic anti-tumor immunity, and optimal gene delivery in an all-in-one therapeutic approach that is delivered intravenously for solid tumors. Their pipeline of "armed" vectors are effective as standalone therapies or as components in combinatorial immunotherapy approaches. Fusix is currently raising a Seed financing round in order to bring its lead development candidate to IND-readiness. They are interested in meeting relevant potential investors and strategic partners.

Website

Biotechnology

Gyre Therapeutics, Inc.

Ticker: NASDAQ: GYRE

San Diego, United States

Wed, 25 Sep · 14:55 – 10:30

Gyre Therapeutics is a commercial-stage biopharmaceutical company headquartered in San Diego, CA focused on the development and commercialization of small-molecule therapeutics with its most advanced programs addressing organ fibrosis and inflammatory diseases.

Gyre’s wholly-owned subsidiary, Cullgen Inc., is a clinical-stage biopharmaceutical company focused on the discovery and development of targeted protein degrader and DAC therapies for critical conditions including cancer, inflammatory diseases, and pain. Cullgen has created a portfolio of highly selective targeted protein degrader and DAC product candidates designed to potently and efficiently eliminate therapeutically relevant proteins in patients.

Website

Laverock Therapeutics Ltd.

United Kingdom

Wed, 25 Sep · 11:50 – 10:30

Laverock Therapeutics is powering the development of disease-responsive advanced therapies through our unique, programmable gene control technology. Our innovative platform harnesses the cell’s natural regulatory mechanisms to deliver programmable and tunable gene control through recoded miRNAs. This enables the development of highly effective medicines with enhanced precision and improved safety profiles. Utilising our platform technologies, we are working to develop the next-generation of advanced therapies, both through our own pipeline – targeting oncology and genetic medicine – and through partnerships. Laverock has a highly experienced leadership team with proven track records in biotechnology, pharma and academia and an exceptionally strong Board. Laverock has raised more than £20m seed funding to date from high-calibre investors including Calculus Capital, Eli Lilly and Company, Mercia Ventures, Maven Capital Partners, Eos Advisory, UK Innovation & Science Seed Fund, Tekfen Ventures and Norcliffe Capital.

Website

BiotechnologyPharmaceuticals/Licensing

LIfT BioSciences Ltd.

London, United Kingdom

Wed, 25 Sep · 15:15 – 10:30

LIfT Biosciences is a biotech bringing to market a first-in-class alpha neutrophil cell therapy that overcomes the limitations of current therapies in solid tumours by destroying tumours both directly and indirectly. LIfT’s Immunomodulatory Alpha Neutrophils (IMANs) turn the tumour microenvironment against the tumour as they recruit the rest of the immune system to destroy the tumour to give a durable response and lasting immunity. The patented breakthrough N-LIfT platform is produced from a patented process using exceptional stem cells (iPSC or HSC), a proprietary enhancement media and genetic engineering (e.g. CARs). The resulting CAR-IMAN cells give cancer patients potentially hundreds of times the cancer killing ability they naturally possess. The company is working with a range of pharmaceutical license partners to develop a portfolio of CAR-IMAN cell therapies to deliver complete remission across all solid tumours before the decade is out. LIfT BioSciences was founded by Alex Blyth following the death of his mother to pancreatic cancer. Technology Immuno-Modulatory Alpha Neutrophil progenitors (IMANs) produced from iPSCs or HSCs using our N-LIfT Platform Stage Pre-clinical work completing, IND filing Focus Solid Tumours with high unmet medical need, starting with SCC-NSCLC, PDAC, HNSCC, UCC Patents 2016 Filing Granted, 6 Patents with FTO Current Raise £25m+ Series A. Pharma license discussions underway. Recent Achievements - Successful production from iPSCs and GMP ready production from HSCs at 10L - Increase T-cell and NK cancer killing 250% in Lab-on-chip tumour model - Unmodified IMANs shows comprehensive solid tumour organoid killing (superior to Keytruda & Abraxane) in NSCLC, PDAC - HER-2 CAR IMANs increase cancer cell killing x5 over the already potent unmodified LIfT IMANs have preclinical validation of all of the characteristics required to overcome the challenges to achieving sustained remission in solid tumours.

Website

Biotechnology

Mabylon AG

Schlieren, Switzerland

Wed, 25 Sep · 11:20 – 10:30

Mabylon, a Swiss Biotech company, builds on its understanding of antibody responses in allergic patients to develop single multispecific antibody molecules that neutralize the allergens and prevent allergic reactions. The first of such products, MY006, is a potent trispecific antibody with half-life extension being developed for peanut allergy and poised to start clinical trials this year. MY006 is designed to provide rapid, safe, and long-lasting protection against peanut allergen exposure. A clinically-validated MoA and a high-yielding and stable product produced by Pfizer, offer together a greatly de-risked development path. The focus of our current fundraising is to show efficacy POC in a phase 1b, an inflection point rendered even more valuable by the adoption of the same endpoint in early and late clinical development. Shortly following, MY010 and MY011 target multiple tree pollens or grass pollens respectively, to relief seasonal allergy symptoms with one single injection before start of the season. Mabylon is positioned to revolutionize the allergy therapeutic landscape by providing effective therapies with simple dosing, allowing allergic patients to regain control of their lives.

Website

Biotechnology

NanoCell Therapeutics, Inc.

Utrecht, Netherlands

Wed, 25 Sep · 12:00 – 10:30

Innovative Non-Viral DNA-based In Vivo Gene Therapy At NanoCell, we envision a future where cell engineering is transformed by pioneering vectors, adept at safely and precisely modifying target cells right within the patient’s body. Our ambition is distinct and audacious. While many in the field focus on viral or RNA-based methods, we uniquely harness the lasting potential of DNA, delivered through our advanced non-viral vector technology. Our goal is to revolutionize current cell and gene therapy practices, primarily CAR and TCR therapy, by eliminating the need for ex-vivo cell manipulations. With the power of DNA and cell-directed lipid nanoparticles (LNPs) – a combination that sets us apart from others – we’re trailblazing a path to durably modify immune-effector cells, such as T cells, directly within patients. This isn’t just innovation—it’s a leap forward, paving the way for cell and gene therapies that are more affordable, scalable, and accessible to individuals across the globe.

Website

Biotechnology

Onward Therapeutics SA

Epilanges, Switzerland

Wed, 25 Sep · 11:10 – 10:30

We are a clinical stage oncology company, focusing on the identification of innovative technologies and the development of truly breakthrough medicines for the treatment of cancer. Our company, led by an experienced team in translational science and drug development, acquires licenses for potential development candidates and invests in partners with transformative technology platforms. We are located at Biopôle, a life sciences campus in Epalinges near Lausanne, Switzerland, we have an affiliate in Paris, France, and also operate from Taipei, Taiwan.

Website

Biotechnology

PDC*line Pharma SA

Liege, Belgium

Wed, 25 Sep · 15:05 – 10:30

Founded in 2014 as a spin-off of the French Blood Bank (EFS), PDC*line Pharma is a Belgian-French clinical-stage biotech company that develops an innovative class of active immunotherapies for cancers, based on a GMP-grade allogeneic therapeutic cell line of Plasmacytoid Dendritic Cells (PDC*line). PDC*line is much more potent than conventional dendritic cell-based vaccines in priming and boosting antitumor antigen-specific cytotoxic T-cells, including the T-cells specific for neoantigens, and is synergistic with checkpoint inhibitors. The technology can potentially be applied to any type of cancer. Following a first-in-human phase I feasibility study in melanoma, PDC*line Pharma focuses on the development of PDC*lung01, a candidate for Non-Small-Cell Lung Cancer (NSCLC) currently in phase I/II trials, and PDC*neo with neoantigens in preclinical development. The company has a staff of 42, with an experienced management team. It has raised close to €61M in equity and non-dilutive funding. In March 2019, PDC*line Pharma granted an exclusive license to the LG Chem Life Sciences company in South Korea and an exclusive option in other Asian countries, for the development and commercialization of the PDC*lung01 cancer vaccine for lung cancer. The total deal is worth €108M, plus tiered royalties on net sales in Asia.

Website

Biotechnology

Pilatus Biosciences SA

Epalinges, Switzerland

Wed, 25 Sep · 10:50 – 10:30

Pilatus Biosciences, a US/Swiss -based R&D startup originating from the Ludwig Institute for Cancer Research (Lausanne), stands on the cutting-edge of developing First-in-Class Biologics, focusing on metabolic checkpoints. Our pioneering approach, backed by the Cancer Research Institute (New York), targeting Immunometabolism triggers immune microenvironment reprogramming to combat cancer.

Website

Biotechnology

PulseSight Therapeutics

Paris, France

Wed, 25 Sep · 16:10 – 10:30

PulseSight Therapeutics, launched early 2024, is an ophthalmology clinical-stage biotech company developing disruptive non-viral vectorized therapies with minimally-invasive delivery technology for AMD. PulseSight has developed a proprietary platform that uses a user-friendly electro-transfection system to deliver DNA plasmids encoding therapeutic proteins into the ciliary muscle to treat eye diseases. The company’s lead program PST-611 in GA is ready to enter the clinic by the end of 2024, whilst its second program, PST-809 in wet AMD is at the very late stage of preclinical IND-enabling studies. Both first-in-class candidates benefit from a solid preclinical package showing their ability to address multiple pathological pathways involved in these two forms of AMD, thus providing a higher efficacy compared to current treatments. First clinical trials have been performed (PST-606 in non-infectious uveitis), showing the very good safety profile of both the plasmid and the delivery procedure as well as the long lasting expression of the therapeutic proteins, allowing to significantly reduce the frequency of injections (4 to 6 months). Pulsesight Therapeutics is managed by a highly experienced executive team and board of Directors; it is financed with seed investment from leading venture capital investors Dominik Escher, PhD, founding partner of Pureos Bioventures and Kostas Kaloulis, PhD, Venture Partner at ND Capital.

Website

Reconnect Labs AG

Switzerland

Wed, 25 Sep · 16:20 – 10:30

Reconnect Labs is a Swiss, clinical stage biotech company developing rapid-acting precision therapeutics for interventional precision psychiatry with the potential to transform the treatment landscape for substance use disorders, post-partum depression, PTSD, and insomnia.

Website

BiotechnologyPharmaceuticals/Licensing

SurgiMab S.A.S.

Montpellier, France

Wed, 25 Sep · 11:00 – 10:30

SurgiMab develops fluorescent molecules that enable real-time visualization of tumors during fluorescence-guided surgery (FGS). These molecules help oncologic surgeons see tumors, micro-tumors, and tumor spread in real-time, reducing both under- and over-treatment. The lead molecule, SGM-101, currently in phase III trials, targets various digestive tumors and has also shown effectiveness in visualizing some lung tumors. SurgiMab is also developing additional products through ongoing collaborations. SurgiMab’s technology uses tumor-specific antibodies linked to a near-infrared fluorescent dye optimized for small tumor nodules. After injection, the molecule targets tumor surface markers, and the fluorescent dye allows visualization of tumors and precise resection by the surgeon. In cases of recurrent cancer, where fibrotic tissue can impair vision, SurgiMab’s molecules help surgeons detect tiny lesions or positive margins that could lead to recurrence if left untreated. Intraoperative imaging with SurgiMab’s molecules improves differentiation between normal and cancer tissue, enabling more precise and radical surgery, better patient outcomes, and reduced healthcare costs.

Website

RISING BIOTECH STARS SESSIONS:

BiotechnologyPharmaceuticals/Licensing

ABYOLO AG

Unterägeri, Switzerland

Thu, 26 Sep · 15:00 – 14:20

ABYOLO: Biosimilars for Everyone, Everywhere ABYOLO is a global biosimilar company committed to making biological medicines accessible and affordable to everyone, everywhere. Our mission is to bridge healthcare gaps through cutting-edge technology and innovative solutions. With an agile, asset-light approach, we swiftly adapt to market changes while maintaining operational efficiency. Headquartered in Switzerland, ABYOLO leverages extensive industry experience and rational thinking to drive the next phase of growth in the pharmaceutical sector. Our team of experts is committed to delivering high-quality, cost-effective biosimilars that meet the needs of patients worldwide. At ABYOLO, we are powered by purpose, and we believe in the power of collaboration and innovation to transform healthcare, navigating regulatory rigidity with a flexible and responsive approach, and revolutionizing industry timing through disruptive technology. Join us on our journey to make biosimilars accessible to everyone, everywhere.

Website

Biotechnology

AIPTO TechBio GmbH

Basel, Switzerland

Thu, 26 Sep · 15:00 – 14:20

Brain tumors, both primary and metastatic, are devastating diseases with a five-year overall survival rate of less than 35%. Despite over 1,000 clinical trials to date, effective treatments remain elusive. The grand challenge is that current drug development and clinical trials are not based on preclinical models that accurately capture disease heterogeneity and most importantly, predict patient responses. AIPTO TechBio addresses this challenge with its revolutionary platform, featuring clinically validated, next-generation patient avatar technology. This technology precisely replicates the cellular and molecular pathology of patients' tumors, including the complex tumor microenvironment. By harnessing the power of generative AI, AIPTO fully exploits patient drug response data, enabling the creation of state-of-the-art AI models. The AIPTO platform represents a paradigm shift in cancer research and drug development, transforming key processes such as target discovery, lead validation, biomarker identification, and patient population stratification. Ultimately, AIPTO is poised to revolutionize drug discovery and deliver groundbreaking therapies for brain cancer patients.

Website

Biotechnology

Akribion Therapeutics GmbH

64673, Germany

Thu, 26 Sep · 15:00 – 14:20

At Akribion Therapeutics we have developed an entirely new modality of cancer cell killing applicable to a broad range of indications, including solid tumors (head and neck, colorectal, pancreatic, lung, brain et al) and hematopoietic malignancies, always characterized by specific RNA biomarker expression.

Website

Biotechnology

Amporin Pharmaceuticals AG

Basel, Switzerland

Amporin is developing the first class of oral drugs that could stop and reverse over 50 deadly degenerative diseases, uniquely by repairing the holes in cell membranes formed by toxic misfolded protein oligomers, allowing diseased cells to fully recover and restore homeostasis. Our vision is to cure 6 major degenerative diseases with a simple pill, starting with Parkinson's disease.

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Biotechnology

Ariya Bio c/o ETH Zürich

Schlieren, Switzerland

Thu, 26 Sep · 15:00 – 14:20

Ariya Bio is a proto NewCo spinning out of the laboratory of the CRISPR-Cas pioneer Prof. Jacob Corn at ETH Zürich. At Ariya Bio, we are pioneering a next-generation gene therapy platform, harnessing blood stem cells, also known as Hematopoietic Stem Cells (HSCs), as living, in-vivo biotherapeutic protein factories. The CRISPR-Cas-based cell and gene therapy platform we are developing is versatile and promises to be easily reprogrammable. We combine two powerful technologies: hematopoietic stem cell therapies and CRISPR-Cas gene editing to develop transformative genomic medicines. More specifically, we use CRISPR Cas to reprogram HSCs to express protein or peptide drugs specifically in red cells and their progenitors. The cargo is expressed either to stay within red cells or to be secreted into the bloodstream, delivering the drug via the vasculature at stable levels, lifelong after one application. This is a novel and transformative treatment modality offering the potential for multiple one-time cures. Our platform is applicable to a wide range of diseases, including Pyruvate Kinase Deficiency, Hemophilia, Pompe disease, and Hemoglobinopathies.

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Biotechnology

Cantoni Therapeutics BV

Leiden, Netherlands

Thu, 26 Sep · 15:00 – 14:20

Cantoni Therapeutics is a private pharmaceutical company based in Leiden, the Netherlands dedicated to the discovery, development and commercialization of best-in-class small molecule inhibitors towards Nicotinamide N-methyltransferase, NNMT, a promising and novel target for cardiometabolic disease, with obesity and its comorbidities selected as lead indication.

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Biotechnology

CaSRevolution S.r.l.

Milano, Italy

Thu, 26 Sep · 15:00 – 14:20

CaSRevolution identified a novel upstream therapeutic target for a disease-modifying approach against Alzheimer’s disease: in fact, pharmacological inhibition and genetic manipulation of its new target - which is promising also because it can be modulated with a small molecule - prevent neurodegeneration in disease-relevant in vitro & in vivo models. The management team and the board of directors have broad experience in company creation & successful exits, neurodegeneration, and drug development. It plans to develop First-in-Class compound(s) to treat early-stage AD, MCI, and dementia due to AD. Therefore, it designed a capital-efficient lead-to-candidate drug discovery plan to be ready for an Investigational New Drug (IND) application on the best candidate within 3 years.

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Biotechnology

Curaxon AG

Oetwil an der Limmat, Switzerland

Thu, 26 Sep · 15:00 – 14:20

Curaxon was founded in 2020 with the goal of developing innovative drugs for severe neurometabolic / neurodegenerative diseases with high medical need and no satisfactory therapeutic options. Company has in-licensed compound CXN-101, a combination of a ProTide iminosugar, to facilitate delivering optimal dose to treat Niemann Pick Type C, Batten Disease and possibly additional rare Lysosomal Storage Disorders with neurological manifestations such as GM1, GM2, Spinocerebellar Ataxia etc. In addition to rare diseases, there is scientific rationale for addressing disease such as Parkinson's GBA1 as well as Alzheimer's Disease. The team works closely with University of Cardiff to in-license additional assets using the ProTide technology.

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Biotechnology

FluoSphera SA

Plan-les-ouates, Switzerland

Thu, 26 Sep · 15:00 – 14:20

FluoSphera is a Swiss biotech developing scalable and transferable human-relevant 3D tissue technologies to improve drug efficacy and safety prediction while reducing reliance on animal testing.

Our proprietary platform combines Pleiad, a portfolio of ready-to-use 3D human tissues, with color-coded multi-tissue systems that recreate complex human biology in a practical, standardized format. Designed for automation and high-content analysis, the platform can be transferred and deployed across laboratories.

FluoSphera is building a portfolio of differentiated applications across efficacy, safety, DMPK-Tox and biomarker discovery. The company has established recurring pharmaceutical customers and signed its first evaluation license agreement with a global CRO, validating the transferability of the platform and its licensing potential. We are scaling through a hybrid business model combining product sales, specialized assays and technology licensing, supported by CRO and distribution partnerships.

We are launching our Series A to accelerate platform automation and transfer, expand the Pleiad product and assay portfolio, grow our international partner network and advance our first human-relevant assay toward regulatory qualification.

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BiotechnologyDrug DeliveryPharmaceuticals/Licensing

Modiblast Pharma GmbH

Deisenhofen, Germany

Thu, 26 Sep · 15:00 – 14:20

Modiblast pursues a novel in vivo cell reprogramming approach to treat hematological malignancies and other cancers. Our proprietary strategy aims to drive leukemia-specific innate & adaptive immune responses and establish memory cells. With our lead pipeline program progressing towards a first clinical trial in AML, we are in the process of raising a seed round. Our long-term vision is to establish a cancer backbone therapy capable of prolonging phases in remission. We aim to enable patients to self-administer this novel drug combination at home, drastically improving their quality of life.

Website

Biotechnology

NatIgGs GmbH

Ulm, Germany

Thu, 26 Sep · 15:00 – 14:20

Protein-misfolding diseases, like Alzheimer's, affect millions of people worldwide, yet effective treatment options remain elusive. NatIgGs GmbH is an Ulm-based biotechnology startup company. Our antibody 'rediscovery' platform integrates natural antibodies' wisdom and optimises antibodies using Artificial Intelligence /Machine Learning technologies to combat protein misfolding diseases. This approach ensures our antibodies can precisely neutralize toxic proteins while sparing healthy ones, offering a superior level of therapeutic specificity.

Website

Biotechnology

PROSION GmbH

Cologne, Germany

Thu, 26 Sep · 15:00 – 14:20

PROSION Therapeutics is an academic biotech spinoff based in Cologne and Berlin, germany. The team pioneered the first approach capable of disrupting the most common communication between disease relevant proteins, making many notorious undruggable targets finally druggable. They are introducing the ProM technology - a new modality capable of transcending the boundaries of small molecules and biologics to engage complex intracellular targets with high specificity.

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Biotechnology

Roca Therapeutics

Nice, France

Thu, 26 Sep · 15:00 – 14:20

Roca Therapeutics is a biotech start-up headquartered in Nice, France, with a groundbreaking ophthalmology program. Our lead candidate, RCT002, is less than 18 months from IND/CTA submission and has shown promising pharmacological data. Developed for topical administration, RCT002 offers a unique pharmaco-economic profile. It complements the current standard of care administered via intravitreal injections (meaning no direct competition), potentially modifying the disease by blocking resistant angiogenesis, oxidative stress, inflammation, and fibrosis. RCT002 is backed by solid translational and mechanistic data. We have designed a robust disease, clinical, and regulatory strategy targeting Radiation-induced Maculopathy (an orphan niche market for accelerated approval) and Diabetic Macular Edema (upside market opportunity). Roca Therapeutics has already secured a international VC (3B Future) for the seed financing who is now looking to co-lead the current Series A funding round to develop RCT002 until the end of Phase 2a (and extend the pipeline). We believe RCT002 represents a compelling investment opportunity.

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Biotechnology

Thabor Therapeutics

Paris, France

Thabor Therapeutics (Thabor Tx) is a biotech company which specializes in the development of an innovative approach to treat patients living with chronic mucosal inflammatory diseases, in particular Inflammatory Bowel Disease (IBD). The company’s goal is to develop first-in-class drugs that specifically target newly identified gut-secreted proteins which disrupt epithelial homeostasis. Based in Paris (F), Thabor Tx was founded in June 2021 and has received seed funding from AdBio partners and Johnson&Johnson Innovation.

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Biotechnology

THERAtRAME S.A.

Liege , Belgium

Thu, 26 Sep · 15:00 – 14:20

THERAtRAME discovers and develops new small molecule inhibitors targeting the tRNA epitranscriptomics to cure patients with untreatable cancer. The THERAtRAME discovery platform integrates unique sets of technologies to discover, develop and position new drugs to beat reluctant cancers. tRNA epitranscriptomics represents a new promising area for drug discovery in oncology. THERAtRAME's ambition is to become a leader in the development and positioning of novel cancer therapies, bringing new hope to patients.

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VIRTUAL SHOWCASES:

Biotechnology

Brenus Pharma

Lyon, France

Brenus Pharma is a French clinical-stage biotech company developing an off-the-shelf platform advancing novel modalities in immuno-oncology. This cutting-edge precision technology mimics tumor protein expression and makes it visible to the immune system, enabling a multi-specific in vivo immune response against evolving tumor cells. www.brenus-pharma.com 

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Medical DevicesCardiology

NovaPump GmbH

Jena, Germany

NovaPump's "PERKAT" plaform (from Jena/ Germany) is the novel percutaneous, self-expanding heart-pump for safe implantations and pulsatile circulation support for acute care patients with clear USP’s (mentioned and cited in leading medical text books and papers, award winning: 1st place at German Medical Award, 1st place at Lothar Spaeth Award from EU Commissioner Guenther Oettinger). So- we are currently rasing our serious round B. Comment: We are former inventors & co-founders of the JenaValve technique, too (the worldwide first technique for selfexpanding TAVI procedures/ to replace the aortic valve percutaneously and wwith a self-expanding NiTi cage; JenaValve was sold to Edwards in 2024 sucessfully. If you want to joyn our investment round possibly (and want to become a part of the word-wide leading cutting-edge technique for acute right heart patients) please get in touch with us (info@novapump.de). Today's mortality rate is for aRHF patients about 50% (30d) so we have the aim & the potential to become a market leader here!

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24th Annual Biotech in Europe Forum

20-MINUTE PRESENTERS:

CNS Pharmaceuticals, Inc.

Herantis Pharma Plc

IAMA Therapeutics S.r.l.

MetrioPharm AG

Nouscom AG

Oryzon Genomics, S.A.

STALICLA SA

TME Pharma

10-MINUTE PRESENTERS:

Ageronix SA

Alder Therapeutics AB

Atamyo Therapeutics SAS

Atopia Therapeutics

Avata Biosciences Ltd.

BiPER Therapeutics

Bloom Science, Inc.

CIS BIOPHARMA AG

éclateral Ltd.

Eleva GmbH

FoRx Therapeutics AG

Fusix Biotech GmbH

Gyre Therapeutics, Inc.

Laverock Therapeutics Ltd.

LIfT BioSciences Ltd.

Mabylon AG

NanoCell Therapeutics, Inc.

Onward Therapeutics SA

PDC*line Pharma SA

Pilatus Biosciences SA

PulseSight Therapeutics

Reconnect Labs AG

SurgiMab S.A.S.

RISING BIOTECH STARS SESSIONS:

ABYOLO AG

AIPTO TechBio GmbH

Akribion Therapeutics GmbH

Amporin Pharmaceuticals AG

Ariya Bio c/o ETH Zürich

Cantoni Therapeutics BV

CaSRevolution S.r.l.

Curaxon AG

FluoSphera SA

Modiblast Pharma GmbH

NatIgGs GmbH

PROSION GmbH

Roca Therapeutics

Thabor Therapeutics

THERAtRAME S.A.

VIRTUAL SHOWCASES:

Brenus Pharma

NovaPump GmbH