Beyond Biotech

→ WHAT IT COVERS Alterna CMO Dr. Narissa Kreyer explains how the company's engineered tRNA therapy AP-003 targets nonsense mutations — a shared error class causing roughly 10% of all genetic disease diagnoses — rather than individual genes, enabling a single molecule to potentially treat dozens of rare diseases simultaneously via basket trial design.

→ WHAT IT COVERS Evox Therapeutics CEO Per London explains how engineered exosomes deliver gene editors directly into the brain, bypassing the blood-brain barrier to target Huntington's disease via MSH3 knockout and ALS via intracisternal administration, with a clinical trial planned for the second half of 2027. → KEY INSIGHTS - **CNS Delivery Route:** For Huntington's, Evox uses convection-enhanced delivery — a one-time neurosurgical procedure that infuses exosome-packaged gene editors...

→ WHAT IT COVERS BioSpain 2026, hosted in Bilbao September 29–October 1 at the Bilbao Exhibition Center, marks the event's first annual edition after two decades of biennial scheduling. Acebio's Stuart Medina outlines the partnering system, four program tracks, international delegation opportunities, and a free Talent Day job fair for biotech professionals.

→ WHAT IT COVERS Nanobiotix founder Laurent Levy explains how hafnium oxide nanoparticles injected directly into tumors amplify radiotherapy efficacy without increasing damage to surrounding tissue, with a phase three head-and-neck trial reading out in early 2026, plus a new Nano Primer platform designed to improve delivery of genetic medicines across oncology and CNS disorders. → KEY INSIGHTS - **Physics vs.

→ WHAT IT COVERS Tony Hixson, Chief Business Officer at Cancer Research Horizons, explains how Cancer Research UK's commercial subsidiary bridges early-stage oncology research to market through spinouts, licensing, and the See Further initiative — a £50M multi-charity joint venture targeting childhood cancer medicines neglected by pharmaceutical companies.

→ WHAT IT COVERS Beijing-based Therona Inc., founded in 2021, has built a circular RNA platform with three active clinical programs — prophylactic vaccine, therapeutic cancer vaccine, and in vivo CAR-T — while remaining largely unknown to Western pharma. CEO Lu Gao outlines the science, manufacturing approach, and dual US-China IND strategy for lead asset TI-0032.

→ WHAT IT COVERS Gene Mack, president and CEO of Gain Therapeutics, explains how GT02287 targets the GBA1-encoded enzyme glucocerebrosidase to potentially modify Parkinson's disease progression rather than manage symptoms. Phase 1b data from 21 patients in Australia shows biomarker reductions in toxic lipid substrates and early functional improvements.

→ WHAT IT COVERS Martin Steiner, Managing Director of DEBRA Research, explains how the German nonprofit bridges early-stage academic EB research and commercial drug development by funding grants, making equity investments in biotech companies, and building dedicated clinical trial infrastructure for a disease affecting roughly 1-in-20,000 people with a life expectancy of 35–40 years.

→ WHAT IT COVERS Bio Europe 2026, returning to Cologne November 9–11 for its 32nd edition, expands from three to seven content tracks, adding regulatory affairs, clinical development, and biomanufacturing programming, alongside a new content-only registration pass designed to bring the full biopharma C-suite into a conference historically dominated by business development professionals.

→ WHAT IT COVERS Andy Parker, CEO of StepPharma, explains how the company's lead drug dancadostat selectively inhibits the CTPS1 enzyme to block cancer cell proliferation across three concurrent clinical trials — relapsed/refractory lymphoma, biomarker-selected solid tumors, and essential thrombocythemia — following a €38M Series C raise. → KEY INSIGHTS - **CTPS1 Selectivity Mechanism:** Dancadostat achieves 1,300-fold selectivity for CTPS1 over CTPS2, meaning healthy tissues can still use...

→ WHAT IT COVERS Arvinas CEO Randy Teal joins Beyond Biotech to discuss the FDA approval of vepdegestrant, the first PROTAC protein degrader to complete Phase 3 trials, explaining how the technology eliminates disease-causing proteins entirely rather than blocking them, and outlining the company's pipeline across oncology and neurodegenerative diseases.

→ WHAT IT COVERS Brandy Wilkinson, CEO of GEM PharmaTech, and neuroscience pipeline leader Ricky Feng explain how the company's library of over 25,000 genetically engineered mouse models addresses neurology's sub-10% clinical success rate by building preclinical tools that mirror human disease biology, biomarkers, and disease staging. → KEY INSIGHTS - **Model selection risk:** Reaching for convenient, overexpressing mouse models rather than disease-relevant ones is a primary driver of CNS drug...

→ WHAT IT COVERS Werner Lanthaler, former Evotec CEO who scaled the company from 200 employees and €40M to 5,000 staff and €800M revenue, shares practical strategies for biotechs attending the BIO International Convention in San Diego, covering preparation, on-site execution, and post-event follow-up to convert meetings into partnerships. → KEY INSIGHTS - **Pre-event targeting:** Biotechs should map their disease area and development stage against the 20–40 pharma companies with publicly known...

→ WHAT IT COVERS Olivia Ryu, Senior Director at Curia, explains how corticosteroid and hormonal therapy APIs — molecules in use for over 70 years — still require highly specialized manufacturing, dedicated segregated facilities, and end-to-end CDMO partnerships to meet growing global demand across aging populations and emerging markets. → KEY INSIGHTS - **Facility Segregation:** Steroid and hormone APIs require physically dedicated, segregated manufacturing plants — not just procedural...

→ WHAT IT COVERS Joachim Eeckhout, cofounder of Labiotech and now co-owner of Knowbio, traces the platform's evolution from a free French-language student blog in 2012 to Europe's leading independent biotech media outlet, covering fundraising, acquisition by Impart in 2021, and the current AI-driven shift in science media. → KEY INSIGHTS - **Media accessibility as a founding strategy:** Labiotech launched in 2012 specifically to solve two concrete problems: biotech media remained in expensive...

→ WHAT IT COVERS Immunic Therapeutics CEO Daniel Witt joins Beyond Biotech on World MS Day to discuss IMU-838, an oral therapy targeting the NR1 nuclear receptor with potential neuroprotective effects in both relapsing and progressive MS, with phase three readouts expected by end of 2025 across 2,221 enrolled patients. → KEY INSIGHTS - **IMU-838 dual mechanism:** IMU-838 inhibits DHODH (the same target as approved MS drug Tecfidera) but also activates the NR1 nuclear receptor, producing direct...

→ WHAT IT COVERS Ochre Bio CEO Quinn Wills and Lexogen CEO Stefan Baj examine why drug discovery fails due to non-human biology models, how they built one of the world's largest human liver functional genomics datasets, and how purpose-designed RNA sequencing data enables AI-driven target discovery for liver disease affecting 1.5 million deaths annually. → KEY INSIGHTS - **Human data gap in liver disease:** Only 40,000 liver transplants are performed globally each year against 1.

→ WHAT IT COVERS Dominic Clark, VP of Technical Operations at IntegraCell (Cryoport Systems), explains why cell therapy programs default to fresh leukapheresis workflows, how late-stage cryopreservation adoption creates costly regulatory and operational problems, and how IntegraCell's automated closed process standardizes starting material handling from early development through global commercialization. → KEY INSIGHTS - **Fresh vs.

→ WHAT IT COVERS Ryan Cavewood, CEO of Labthread, draws on a decade running Oxgene (acquired by Wuxi Advanced Therapies in 2021) to explain how fragmented lab tools—scattered across Excel, email, and paper notebooks—erode reproducibility, destroy institutional knowledge, and consume scientist time that should go toward research. → KEY INSIGHTS - **Knowledge retention cost:** When a scientist leaves after two to three years, all their work risks becoming inaccessible if stored in personal folder...

→ WHAT IT COVERS Stanford bioengineering professor Stanley Qi, founder of EpicBio, explains how the company's GEMS platform uses a miniaturized Cas protein — one-third the size of Cas9 — to perform reversible epigenetic editing without cutting DNA, with lead program EPI-321 targeting FSHD muscular dystrophy in an upcoming Phase 1 trial. → KEY INSIGHTS - **Mutation-agnostic targeting:** Epigenetic editing can treat diseases with hundreds of distinct mutations in a single gene — like cystic...