In the field of biotechnology, breakthroughs in treating neurological conditions have come a long way. Gene therapy, a method that modifies the genetic material inside cells, has stood out as a promising option, particularly for patients battling hard-to-treat disorders. EpilepsyGTx, a biotech startup from Cambridge, has propelled its work in this area forward by securing €28 million in funding for its lead program aimed at severe epilepsy. This development is exciting for the startup world, but it also paints a larger picture of where the industry is heading.
The funding that made it possible
When new funding rounds are announced, I like to analyze what the numbers actually bring to the table. For EpilepsyGTx, this €28 million infusion, backed by XGEN Venture, the British Business Bank, and a major global pharmaceutical player, is more than fuel for clinical trials. It’s a signal that investors are turning their focus not just to the health outcomes but to treatments that could eliminate long-term dependency on medication. And let’s be honest, any innovation that targets neurological disorders with this level of precision is bound to reshape approaches in healthcare.
So, what does this startup have that caught investors' eyes? The answer lies in EPY201, a gene therapy tailored to focal refractory epilepsy (often shortened to FRE), a condition that leaves millions of patients with seizures, even after trying multiple medications. This therapy, using adeno-associated viral (AAV) vectors, targets the precise area in the brain triggering the seizures. Smart, right? A one-time, minimally invasive treatment could free FRE patients from decades of treatments that barely work.
EpilepsyGTx’s journey and strategy
EpilepsyGTx operates as a spinout of the UCL Queen Square Institute of Neurology, a clear advantage in a field built on academic research. By partnering closely with universities, startups can leverage talent and ready-made research infrastructure. But while many startups fall into a too-academic-model trap, this company has made a move that’s often overlooked: translating research into commercially viable products.
Let’s get specific. Instead of aiming to replace general epilepsy treatments, EpilepsyGTx is narrowing its focus. FRE affects a smaller population than general epilepsy, but it’s a desperate market. Their move also sidesteps bloated competition from large pharmaceutical companies focused on broader drug categories. For founders wondering whether niches are worth it, yes, they are. Especially when solving a real, measurable pain.
Trends shaping the space
There’s been a notable shift in investment into biotech over the past two years. Neurological disorders have a reputation for being hard to treat, and for decades, they were underfunded because of the long runway to see clinical results. But not anymore. In Europe, we’re seeing startups like EG 427 and TRIMTECH Therapeutics, which have recently raised over €27 million and €28.6 million, respectively. Both are investigating treatments related to the brain or central nervous systems. The demand from an aging population, combined with better research tools, has turned biopharma into one of the hottest sectors to watch.
What’s particularly interesting is how venture capitalists are pooling their bets here. The British Business Bank’s €12 million stake in EpilepsyGTx’s Series A is an example of institutional support for taking more calculated product development risks. It’s not an act of charity; it’s recognizing that startups with well-defined, patient-focused solutions have a strong chance of crossing the regulatory hurdles biotech is infamous for. Investors take note: there’s real ROI waiting.
How startups can do better: Avoiding common pitfalls
Founders in any medical or biotech field face a tough road ahead, but here’s what can sink even the most promising project:
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Overpromising early results: Claiming too much during preclinical stages can bite you later. Investors need clear, realistic timelines.
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Ignoring commercialization early on: Research without an application path ends up in the proverbial "academic graveyard." Start speaking to regulators and insurers from day one.
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Overlooking patient diversity: Neurological disorders often have complex presentations across age groups, genders, and genetic backgrounds. Building a trial population representative of real-world patients is non-negotiable here.
Applying lessons from EpilepsyGTx: A guide for aspiring founders
As someone who’s worked around startups and innovation hubs for most of my career, watching how biotech teams navigate funding stages is fascinating. Here’s what I recommend copying from this case:
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Identify a specific unmet need: EpilepsyGTx didn’t go after “epilepsy” broadly. By focusing narrowly on FRE, they honed their pitch.
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Leverage academic partnerships: University spinouts often struggle with messy ownership models, but EpilepsyGTx clearly put in the work to get clean IP (intellectual property) that investors could trust.
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Prioritize investors with aligned expertise: Generalist VCs are helpful, but filling your round with sector-focused funds adds real value. XGEN Ventures, for instance, has expertise in disorder-specific therapies.
What entrepreneurs often miss when chasing biotech valuations
Let me give you an actionable reality check. Biotech valuations tend to balloon during medical advancements, but they burst if not backed by sound science. If you’re building for the healthcare sector under any vertical (AI, gene therapy, medical devices), public trust in your narrative can make or break you. EpilepsyGTx’s approach was to align their messaging tightly with science while showing it could work for patients who’ve spent thousands on ineffective treatments.
Marketing also matters, a lot. Entrepreneurs often think great science markets itself. It doesn’t. Patients, policymakers, and insurers all need a clear story about how you’re reducing pain points. Focus just as much on storytelling as you do on the therapeutic mechanism.
Wrapping it up: A win for science and business
EpilepsyGTx’s success isn’t just a win for advanced treatments; it’s a clear example of what happens when medical startups stay nimble but precise. While big pharmaceutical companies move slowly, startups closest to research and patients have the edge in innovating.
For the business-savvy among us: the global gene therapy market is expected to reach billions in the coming years. Whether as a founder, investor, or advisor, now’s a great time to engage with science-backed companies creating life-changing solutions. Just do what EpilepsyGTx did, focus on the real problem, build trust, and invest in your niche while bigger players wrestle with broader markets. That’s where startups shine.
FAQ
1. What is EpilepsyGTx, and what does it specialize in?
EpilepsyGTx is a Cambridge-based biotech startup focusing on developing gene therapies to treat focal refractory epilepsy (FRE), a severe neurological condition characterized by treatment-resistant seizures.
2. How much funding has EpilepsyGTx raised recently?
EpilepsyGTx secured €28 million ($33 million) in Series A funding to advance its gene therapy program, EPY201, into Phase 1/2a clinical trials. Read more about the funding on EU Startups
3. What is EPY201, and how does it work?
EPY201 is an adeno-associated viral (AAV) gene therapy designed to deliver genetic material directly to the brain's seizure focus. This minimally invasive therapy aims to reduce neuronal hyperexcitability and stop seizures. Read more about EPY201 development
4. Who are the major investors in the €28 million funding round?
Investors in this Series A round include XGEN Venture, the British Business Bank (contributing €12 million), and an anonymous global pharmaceutical company. Check out British Business Bank's involvement
5. What makes EpilepsyGTx’s approach unique within the market?
EpilepsyGTx focuses on a highly targeted treatment for focal refractory epilepsy, a smaller but underserved patient population, by leveraging a one-time gene therapy solution rather than lifelong medication or invasive surgery.
6. How does EpilepsyGTx benefit from its academic partnerships?
EpilepsyGTx is a spinout of the UCL Queen Square Institute of Neurology, which provides a strong foundation in research, infrastructure, and talent to refine its therapies for clinical applications. Read more about EpilepsyGTx’s academic origins
7. How does the funding landscape look for neuroscience-related biotech startups?
The field is experiencing a surge in investments, with companies like TRIMTECH Therapeutics (€28.6M) and EG 427 (€27M) raising significant funds for neurological disorder treatments. Explore Europe’s funding trends
8. What challenges do medical startups like EpilepsyGTx face?
Key challenges include overpromising early results, failing to consider commercialization paths, and neglecting diversity in clinical trials. Overcoming these pitfalls is critical for success.
9. Why is precision neurology important in biotech?
Precision neurology, exemplified by therapies like EPY201, offers customized solutions targeting specific neurological conditions, improving efficacy and reducing side effects compared to generalized treatments.
10. What is the significance of this funding round for the biotech industry?
EpilepsyGTx’s funding underscores the increasing trust from investors in gene therapies and the biotech sector’s ability to address complex neurological disorders with innovative solutions.
About the Author
Violetta Bonenkamp, also known as MeanCEO, is an experienced startup founder with an impressive educational background including an MBA and four other higher education degrees. She has over 20 years of work experience across multiple countries, including 5 years as a solopreneur and serial entrepreneur. Throughout her startup experience she has applied for multiple startup grants at the EU level, in the Netherlands and Malta, and her startups received quite a few of those. She’s been living, studying and working in many countries around the globe and her extensive multicultural experience has influenced her immensely.
Violetta Bonenkamp's expertise in CAD sector, IP protection and blockchain
Violetta Bonenkamp is recognized as a multidisciplinary expert with significant achievements in the CAD sector, intellectual property (IP) protection, and blockchain technology.
CAD Sector:
- Violetta is the CEO and co-founder of CADChain, a deep tech startup focused on developing IP management software specifically for CAD (Computer-Aided Design) data. CADChain addresses the lack of industry standards for CAD data protection and sharing, using innovative technology to secure and manage design data.
- She has led the company since its inception in 2018, overseeing R&D, PR, and business development, and driving the creation of products for platforms such as Autodesk Inventor, Blender, and SolidWorks.
- Her leadership has been instrumental in scaling CADChain from a small team to a significant player in the deeptech space, with a diverse, international team.
IP Protection:
- Violetta has built deep expertise in intellectual property, combining academic training with practical startup experience. She has taken specialized courses in IP from institutions like WIPO and the EU IPO.
- She is known for sharing actionable strategies for startup IP protection, leveraging both legal and technological approaches, and has published guides and content on this topic for the entrepreneurial community.
- Her work at CADChain directly addresses the need for robust IP protection in the engineering and design industries, integrating cybersecurity and compliance measures to safeguard digital assets.
Blockchain:
- Violetta’s entry into the blockchain sector began with the founding of CADChain, which uses blockchain as a core technology for securing and managing CAD data.
- She holds several certifications in blockchain and has participated in major hackathons and policy forums, such as the OECD Global Blockchain Policy Forum.
- Her expertise extends to applying blockchain for IP management, ensuring data integrity, traceability, and secure sharing in the CAD industry.
Violetta is a true multiple specialist who has built expertise in Linguistics, Education, Business Management, Blockchain, Entrepreneurship, Intellectual Property, Game Design, AI, SEO, Digital Marketing, cyber security and zero code automations. Her extensive educational journey includes a Master of Arts in Linguistics and Education, an Advanced Master in Linguistics from Belgium (2006-2007), an MBA from Blekinge Institute of Technology in Sweden (2006-2008), and an Erasmus Mundus joint program European Master of Higher Education from universities in Norway, Finland, and Portugal (2009).
She is the founder of Fe/male Switch, a startup game that encourages women to enter STEM fields, and also leads CADChain, and multiple other projects like the Directory of 1,000 Startup Cities with a proprietary MeanCEO Index that ranks cities for female entrepreneurs. Violetta created the "gamepreneurship" methodology, which forms the scientific basis of her startup game. She also builds a lot of SEO tools for startups. Her achievements include being named one of the top 100 women in Europe by EU Startups in 2022 and being nominated for Impact Person of the year at the Dutch Blockchain Week. She is an author with Sifted and a speaker at different Universities. Recently she published a book on Startup Idea Validation the right way: from zero to first customers and beyond, launched a Directory of 1,500+ websites for startups to list themselves in order to gain traction and build backlinks and is building MELA AI to help local restaurants in Malta get more visibility online.
For the past several years Violetta has been living between the Netherlands and Malta, while also regularly traveling to different destinations around the globe, usually due to her entrepreneurial activities. This has led her to start writing about different locations and amenities from the POV of an entrepreneur. Here’s her recent article about the best hotels in Italy to work from.
About the Publication
Fe/male Switch is an innovative startup platform designed to empower women entrepreneurs through an immersive, game-like experience. Founded in 2020 during the pandemic "without any funding and without any code," this non-profit initiative has evolved into a comprehensive educational tool for aspiring female entrepreneurs.The platform was co-founded by Violetta Shishkina-Bonenkamp, who serves as CEO and one of the lead authors of the Startup News branch.
Mission and Purpose
Fe/male Switch Foundation was created to address the gender gap in the tech and entrepreneurship space. The platform aims to skill-up future female tech leaders and empower them to create resilient and innovative tech startups through what they call "gamepreneurship". By putting players in a virtual startup village where they must survive and thrive, the startup game allows women to test their entrepreneurial abilities without financial risk.
Key Features
The platform offers a unique blend of news, resources,learning, networking, and practical application within a supportive, female-focused environment:
- Skill Lab: Micro-modules covering essential startup skills
- Virtual Startup Building: Create or join startups and tackle real-world challenges
- AI Co-founder (PlayPal): Guides users through the startup process
- SANDBOX: A testing environment for idea validation before launch
- Wellness Integration: Virtual activities to balance work and self-care
- Marketplace: Buy or sell expert sessions and tutorials
Impact and Growth
Since its inception, Fe/male Switch has shown impressive growth:
- 5,000+ female entrepreneurs in the community
- 100+ startup tools built
- 5,000+ pieces of articles and news written
- 1,000 unique business ideas for women created
Partnerships
Fe/male Switch has formed strategic partnerships to enhance its offerings. In January 2022, it teamed up with global website builder Tilda to provide free access to website building tools and mentorship services for Fe/male Switch participants.
Recognition
Fe/male Switch has received media attention for its innovative approach to closing the gender gap in tech entrepreneurship. The platform has been featured in various publications highlighting its unique "play to learn and earn" model.