Tag: marketaccess

10 March 2026

Question:

BIO.NRW sits at the intersection of science, start-ups, industry and policy. In your view, what is still underestimated when it comes to translating excellent science into sustainable biotech companies?

Answer:

For me, this question has two dimensions. First, what does it actually take to make translation successful? And second, how do we define sustainable success?

What we consistently take away from many conversations and experiences is that it starts with the right people. The team is absolutely critical. That is something we hear from investors again and again. Then you need the right infrastructure. And today, infrastructure does not just mean lab space or bench space. It means comprehensive support across all relevant areas, such as HR, IP protection, administration, legal matters, funding, and operational guidance.

The third point is capital. Without money, nothing happens.

The interesting part is the word “sustainable.” Founding a company and developing it to an exit is the normal life cycle of many biotech companies. From a technology perspective, that may be sustainable, because the innovation is absorbed into larger structures and taken forward there. But from the perspective of a region like NRW, that is not yet the kind of sustainable development we would like to see.

We would like to see more companies that continue to grow locally, raise additional financing rounds, build broader pipelines, and develop beyond an early exit. That is much rarer and for that, many factors need to come together.

And one thing should never be forgotten: in pharma, development risk always remains. If something goes wrong in Phase 2, a company can very quickly reach its limits. Those inherent risks can never be fully eliminated, and they always stand in the way of truly sustainable biotech development to some extent.

System Friction

Question:

Where do you currently see the biggest systemic friction points that slow down the translation of research into internationally competitive biotech companies?

Answer:

In Germany — and also in NRW — we clearly have challenging framework conditions, especially on the regulatory and administrative side. The whole area of approvals, permits, and interactions with authorities is a real problem. In some individual cases, it is even existential, including for larger companies. That is an area where we clearly have work to do.

The second major point is capital. Compared with the US, there is significantly less money in the market, on a completely different scale. We can partly compensate for that through strong science, or by pushing development somewhat further within academia, but fundamentally it remains a structural disadvantage.

In the seed and pre-seed space, we have fewer problems today than in the past. There are good examples of strong early-stage financings. But those are still more the exception than the rule. Overall, capital availability remains a limiting factor.

Global Positioning

Question:

If you compare NRW with international hubs such as Boston, Cambridge or Basel: where do you see an underestimated structural strength and where do you see a weakness we still have not addressed sufficiently?

Answer:

A clear strength of NRW is its diversity. If you look at NRW as a whole, structurally we are not as far away from established clusters as people sometimes assume. The bigger issue is that NRW is not perceived as one clearly visible, concentrated location.

We have the densest higher-education network in Europe, with around 70 universities and universities of applied sciences. That is a tremendous asset. We have strong locations across many disciplines: MedTech and engineering in Aachen, strong cancer research, gerontology in Cologne, and overall a very broad scientific base. That diversity is a real advantage.

The weakness is that this strength is not sufficiently visible. We are not concentrated in one place in the same way Basel or Cambridge are. That makes perception, branding, and international positioning more difficult. On top of that, we have less capital in the market and no comparable Big Pharma anchor like Basel.

Perhaps a certain reluctance around risk is part of it as well. When resources are more limited, people tend to focus more on de-risking. That is understandable, but at some point, you also have to be willing to take calculated risks if you want to compete internationally.

Valley of Value

Question:

Many companies survive the early phase, but struggle to build sustainable value. In your experience, where does the German biotech system lose the most value and why?

Answer:

I strongly agree that the core problem today is no longer the very early stage. Pre-seed and seed are functioning much better than they used to. The major challenge begins with follow-on financing.

If a company is to be built in a truly sustainable way, it often needs much larger financing rounds, not just five or ten million, but potentially fifty or eighty million. That is exactly where things become difficult.

The problem has actually become more severe in recent years because pharmaceutical companies have become more risk-averse. Partnerships and acquisitions happen later. Pharma wants to see more data before making larger decisions. The consequence is that biotech companies have to keep developing longer on their own and that additional development runway requires time, people, and capital.

A good example is the changing value-creation logic in early pharma. In the past, certain programs could be licensed or sold much earlier. Today, that is often no longer enough. More data, more structure, and more team building are required. That means new companies have to be created to carry out that development work in the first place. This is exactly where value is lost if capital, resources, and time are insufficient.

And then there is bureaucracy. Regulatory requirements and administrative burdens consume resources and slow progress. That, too, ultimately destroys value.

Pharma–Startup Interface

Question:

Large pharmaceutical companies often say they want to work with start-ups earlier. In your view, what do start-ups most often misunderstand about pharma and what does pharma underestimate about early biotech companies?

Answer:

One important issue is that start-ups often do not know the right contacts or decision-making pathways inside large pharmaceutical organizations. Especially in the beginning, there is often a certain hesitation to approach pharma at all. People do not know whom to speak to, whether there is any interest, or how these organizations actually work and everything can appear very closed and highly proprietary.

That is exactly why clusters and exchange platforms are so important. They create the conditions for contacts to emerge, and they help founders understand how pharma thinks, what pharma is looking for, and how collaboration can actually be initiated. Without such structures, that bridge is often missing.

At the same time, start-ups often worry that if they engage too early, their IP or USP may be diluted or absorbed. That concern is understandable. Which is why formats that build trust and mutual understanding are so important.

Programs such as JLABS or similar initiatives from larger companies can help lower exactly these barriers. Such formats create exchange, facilitate access, and encourage a much better understanding on both sides. In successful clusters such as Basel, you can clearly see how important these structured spaces for interaction are.

Policy vs. Execution

Question:

Germany has many programs, grants and initiatives. If you could change one policy lever tomorrow to improve execution speed in biotech, what would it be?

Answer:

I would especially like to see more funding in later technology readiness level (TRL) stages, meaning larger ticket sizes, larger funding volumes, and not just many small projects. For pharma and biotech in particular, this is crucial, because capital requirements are shifting further downstream as partnerships happen later.

If pharma comes in later and follow-on financing is missing at the same time, then in principle public funding could help close part of that gap. But those instruments are largely missing, or only available in very selective cases.

We have seen some individual programs with larger volumes, but mostly in very specific structural funding contexts. If there were a strategic will to do so, one could create stronger, more selective programs that move the most promising companies forward more quickly with meaningful funding volumes. From my perspective, that would be a very important lever.

Talent & Leadership

Question:

Scientific excellence is there, but experienced biotech leadership is still scarce. How critical is operational and strategic maturity compared with science and how can clusters like BIO.NRW help close that gap?

Answer:

It is essential. Science alone is not enough. Experienced leadership, operational maturity, and strong decision-making capability are absolutely central.

What we can do — and what we are already doing — is provide support through coaches, mentors, and experienced sparring partners. Programs like GeneNovate show how important those structures are.

What matters, however, is a systemic approach: start-ups should, if they want it, have early access to professional support tailored to their individual stage and needs. That can range from time-limited coaching to more intensive hands-on support. Fundamentally, we need these support structures on a permanent basis.

Founder Mindset

Question:

If you could give founders in NRW one uncomfortable message early on, what should they stop doing, even if it feels scientifically right?

Answer:

Founders need to become more focused. They need to think much more from the product side and from the path to the product, not only from the scientific idea.

A common mistake is to get lost in maximum market potential, global opportunity narratives, or a large number of possible applications. But what investors want to understand is this: what is the actual product? How do you get there? And where can you create real traction or cash-flow relevance first?

Trying to address everything at once does not work. Focus is critical.

Closing Question

Question:

If you look five to ten years ahead: what would real success for BIO.NRW look like, not in KPIs or funding volumes, but in outcomes that truly matter for patients, companies and the ecosystem?

Answer:

For me, real success would mean a vibrant, broad biomedical pipeline coming out of NRW, with meaningful developments in areas such as ATMPs, cancer, Alzheimer’s and other biopharmaceutical fields and above all successful commercializations that originate here and actually reach patients.

It is not about limiting that vision to individual indications. What matters is that we see a number of real success stories: programs and companies that advance clinically and commercially, become visible beyond the region, and create momentum for the ecosystem as a whole.

Of course, the benefit to patients is always the ultimate goal. But such success stories are also critical in making NRW visible as a strong biotech location. And I am optimistic that in five to ten years, we will see significantly more examples of that kind.

16 December 2025

1. The M&A environment

Jens: How would you describe the current state of pharma and biotech M&A? Are we returning to pre-2022 dynamics, or has something fundamentally shifted?

Alan: The M&A landscape in biopharma is primarily driven by upcoming losses of exclusivity over the next five to ten years. Big pharma needs to replenish the top line because several important products are going away.

Valuations are also relatively favorable, which is why we’re seeing many bolt-on acquisitions. Buyers tend to focus on later-stage assets, either commercial products or programs with established proof of concept, so they can get to market fairly quickly.

There is also a focus on platform technologies: either because a product can expand into multiple indications, or because the underlying technology can be applied across other parts of the business.

Overall, there’s renewed interest in the sector, and that’s part of why sentiment has improved. However, biotech still faces fundamentals it hasn’t solved: the cost of developing drugs hasn’t improved in years, timelines remain long, and risk hasn’t diminished. At the same time, price control pressures are increasing, and competition from China has not been fully addressed, particularly in the U.S., and to some extent in Europe as well. So yes, there is breathing room due to M&A activity, but the industry should use this period to transform and become more efficient, so it comes out of this window on a stronger foundation.

Jens: I agree. Years ago, when I was co-founding biotech companies, platform technologies weren’t particularly attractive to investors, people focused on single drugs. That seems to have changed, and platform thinking is now also used to de-risk and broaden the opportunity.

Alan: Exactly. There’s a growing realization in large pharma that you need access to large markets. Obesity illustrates that. Look at the valuation multiple of Eli Lilly versus other big pharma, much higher, largely driven by the perceived long-term value of their obesity franchise.

That is pushing companies to prioritize very large opportunities. A product that will never exceed a billion dollars can be hard to justify in a very large organization. As a result, we expect consolidation in rare disease as big pharma exits. Consolidation in specialty pharma is also ongoing.

It will be interesting to see how biotech positions itself to be attractive to different pools of buyers.

2. What makes biotech attractive today

Jens: One major shift I see is evidence-based development: having a clear plan not only for the clinical trial, but also for biomarkers, endpoints, and early payer discussions, so pharma sees lower risk in licensing or acquiring. Ten years ago, many of us weren’t thinking about payers early.

Alan: I agree. One hope I have is that we reach the end of what I call the “biotech mafia”, financial engineering around products or platforms that are overly complex for the public to evaluate, where IPOs were used as a proof point for early exit, leaving less sophisticated investors holding the risk.

Greater access to information may reduce that. The IPO window still hasn’t truly reopened, and we probably need additional interest rate cuts before it does. But with more transparency, capital allocation should improve, though we’ll see.

3. Acquisition logic: diligence, CMC, and market reality

Jens: Today, does the “whole package” matter more, CMC readiness, clinical development quality, strong proof of concept? Has acquisition logic changed compared with 20 years ago?

Alan: These have always been the basic building blocks. If you’re spending hundreds of millions, or even ten million, you should do thorough due diligence to understand risk across all dimensions.

Yet deals still happen under urgency where diligence is limited, and that often comes back to hurt the buyer. We’ve seen situations where buyers paid extremely high multiples and later realized the assets couldn’t be leveraged the way they expected. Those are failures of decision-making and diligence.

Also, especially on the investment side, understanding the ultimate market has become far more important. The earlier you understand payer reality, the better your decision-making becomes.

The old assumption, “if the science is good, someone will figure out how to sell it”, is changing. Commercial practices have tightened. Products must demonstrate meaningful value to patients, and payers need to agree.

Still, we regularly see investments where this market work hasn’t been done. Teams focus on “what’s the next clinical trial” instead of asking: what will competitiveness look like in five or ten years? Who else is coming? What alternative mechanisms or technologies could challenge this? What are payers thinking? Unfortunately, this isn’t systematic enough.

4. Acquisition vs. interest

Jens: What triggers serious acquisition or partnering interest today? What makes a company “deal-ready”?

Alan: It starts with strategy. If you have a clear strategy, you understand what growth you need, at what pace, and whether you need inorganic options versus internal build.

Then there are events you can’t foresee, like a Phase III failure, that suddenly force action. A company may need to acquire or license a stopgap to stabilize while conditions change. That remains a key driver.

The competitive landscape matters on both the buy side and the sell side. Companies ask whether they can realistically be among the top three in an area. If they can’t, it may no longer make sense to keep investing, and divestment becomes the rational move.

For example, we’re working with a company that developed a platform and initially pursued immunology assets, but realized the platform has greater potential in metabolic disease. They refocused resources toward metabolic development and are now selling a portfolio of five early-stage immunology assets, interesting assets, just not strategic for them.

There are also cases driven by external pressure. If investors push for improved profitability or margins, companies may divest lower-margin businesses to improve overall economics and, by extension, valuation multiples.

So it’s a combination of long-term shareholder value creation, strategic discipline, and not missing emerging hyper-growth opportunities. A strong example is Eli Lilly’s GLP-1 strategy: many assume it “just happened,” but leadership has described having a clear strategy in 2017. That kind of foresight is impressive.

5. Why deals stall or fail

Jens: Why do some deals stall or fail, and how can leaders overcome those issues?

Alan: Most often, deals fail due to lack of alignment at the decision-maker level. A mentor once told me: “You’re the conductor on the train, make sure nobody gets off.” That’s accurate. Deals can derail easily.

It’s always easier to say “no” than to say “how do we make this work?” You want people who focus on making the deal work in a way that creates value for both organizations.

Alignment among decision-makers on both sides is critical. You need access to the real decision-makers and confidence that your counterpart truly has their ear. You need active communication back and forth, and ideally you become allies, each working internally to keep your respective organizations aligned. Many deals fail because that alignment work doesn’t happen.

6. Global perspective

Jens: Do you observe differences in how U.S., European, and Asian companies approach M&A valuation and risk? What should CEOs adapt when engaging across regions, especially given China’s competitiveness?

Alan: Culture is very different, and you have to be aware of that.

In Asia, deals typically start with the relationship. You build trust first, and then you do the deal.

In the U.S., it’s often more transactional: you can go straight to the terms and the numbers. You don’t need to be friends to do business.

That affects meeting dynamics. A hard-charging American style can create distrust in Asia if the relationship foundation isn’t there yet. Trust needs to be built.

On risk: in general, Americans are less risk-averse, sometimes because leaders in public companies may only have a three-to-five-year window in their role and want to show impact, transactions included.

Many Asian companies, often family-owned or operating with multi-generational horizons, and to some extent Europe as well, think longer-term. They are more cautious because decisions affect long-term sustainability and, in some cases, future generations.

The downside is that greater caution can mean missing opportunities. It’s the classic trade-off: high risk, high reward.

7. Deal readiness for 2026

Jens: What recommendations would you give CEOs preparing for M&A readiness in 2026? Where should they invest time and resources early?

Alan: The fundamentals are the same: clear data, a well-run operation, and clean financials.

In every deal, there is usually a “skeleton in the closet” somewhere. The key is to surface it, not hide it – and have a mitigation plan. Show the potential acquirer you understand the issue and are actively managing it.

Valuation sophistication is increasing. With scenario tools, including AI-based approaches, you should have a strong understanding of what your company is worth and why.

Clarity of purpose also matters. If you decide to sell, be decisive. Not “we’re exploring,” but: we’ve done the work, we know our value, we know where it comes from, we have a timeline, and we are executing. That gives buyers confidence their resources won’t be wasted.

Finally, be collaborative and transparent. Your fiduciary responsibility is to maximize value, but you do that by being clear about your model and assumptions, not by hiding or embellishing. You are who you are as an organization.

The best way to maximize value is to attract multiple credible parties and create competition. Transparency helps build buyer confidence and broadens the pool. To use an analogy: if you sell a house and show everything openly, more people can get comfortable, and you create competition. Too often companies hide issues, end up with only one buyer, and then fail to maximize value.

8. What will shape the next wave of pharma M&A

Jens: Final question: what scientific fields or business models will shape the next wave of pharma M&A?

Alan: AI will drive a lot of transformation. We started this conversation with the need for biotech to become more efficient. AI has the potential to streamline multiple verticals, discovery, manufacturing, regulatory, and clinical, so the process of bringing a drug to market becomes faster and less costly.

Historically, timelines expanded dramatically. There was a time when companies could bring drugs to market far faster than today. While safety and regulatory standards have evolved, we now finally have technologies that could compress timelines and reduce cost.

That enables new business models. If time-to-market and cost-to-market decline, pricing logic may evolve as well. AI can also support greater precision, targeting specific genotypes or subpopulations rather than broad, less efficient approaches.

Today there are roughly 2,000 pharmaceutical products targeting a few hundred mechanisms of action. There is heavy concentration around known biology, intense competition, and significant waste, especially when you’re not among the top three in a category. Over time, AI could help reduce redundancy and improve capital allocation.

This will take time – likely ten to fifteen years – but it’s coming.

Beyond AI, prevention could become more important. Pharma is largely treatment-oriented today; a more prophylactic model could grow.

Finally, global commercialization and collaboration are important. The current system, every country having its own regulatory agency, creates artificial silos and inefficiencies. Greater harmonization would be beneficial.

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Jens: Thank you for your time and input.

13 November 2025

1. Progress of Genethon’s Gene Therapy Programs

Q: How are Genethon’s programs progressing, especially in neuromuscular diseases?

A: Genethon is currently advancing a robust pipeline of gene therapy programs with a strong focus on neuromuscular and metabolic disorders. One of the most significant recent achievements is the progression of the Duchenne muscular dystrophy (DMD) program, GMT-0004, into Phase 3. This represents the most advanced Duchenne gene therapy program in Europe. Early data from the dose-escalation cohorts show not only biochemical improvements, such as a roughly 75% decrease in circulating creatine kinase (CK), but also stabilization of motor function—an important indicator given the relentless progression of Duchenne.

Genethon is also progressing multiple additional neuromuscular programs toward pivotal readiness. In Crigler–Najjar syndrome, one of the longest-running clinical efforts, several treated patients have now been living for almost five years without requiring phototherapy. This represents a profound improvement in quality of life and a durable therapeutic effect.

2. Clinical Impact and Dose Advantages

Q: What differentiates Genethon’s Duchenne gene therapy from other approaches?

A: A key differentiator of GMT-0004 is its dose efficiency. Compared to other leading programs in the Duchenne space, Genethon administers vector at much lower doses while achieving meaningful and durable clinical benefits. Specifically, the program uses a dose approximately four times lower than Sarepta’s SRP-9001 therapy and nearly seven times lower than RGX-202 from Regenxbio/Solid Biosciences. Lower dosing is clinically and commercially significant: it reduces the risk of immune-mediated toxicities, improves overall safety margins, and eases manufacturing demands, all of which contribute to a more scalable and sustainable therapy.

The differentiation is further reinforced by the fact that Sarepta’s Phase 3 trial did not achieve statistical significance on its primary endpoint. Against this backdrop, the stable motor function and biomarker improvements observed in Genethon’s early cohorts — coupled with the consistency of response across multiple clinical centers — provide strong confidence in the program’s therapeutic potential.

3. Challenges in the One-Shot Gene Therapy Model

Q: Why is the one-time gene therapy business model difficult to sustain?

A: While the vision of a one-time curative therapy has driven much of the field’s early excitement, the business model underlying a single-administration therapy is proving difficult to sustain. Because a one-time treatment must recoup the full cost of research, development, manufacturing, and risk within a single dose, pricing pressures inevitably rise to levels that strain payer budgets.

In addition, the biological and clinical reality of many diseases complicates the concept of a single-dose cure. Tissues regenerate, patients grow (especially pediatric patients), vector expression can decline over time, and the disease may continue progressing. These practical considerations create demand for re-dosing — something that most current AAV-based therapies cannot support due to long-lasting immunity.

The future of gene therapy is likely to shift toward re-dosable or chronic administration. This transition is being enabled by innovations such as lipid nanoparticles (LNPs), next-generation capsids with reduced immunogenicity, and improved immune-modulation strategies that may one day allow safe AAV re-dosing. Such solutions would shift gene therapy economics toward more traditional pharmaceutical models.

4. Ultra-Rare Diseases and New Funding Models

Q: What makes ultra-rare diseases especially challenging?

A: Approximately 85% of rare diseases fall into the category of ultra-rare disorders, each affecting fewer than one in 100,000 individuals. While the scientific rationale for treating these conditions is often strong, they are generally unattractive markets for pharmaceutical companies and venture investors, who require larger patient populations for meaningful return on investment.

For patient organizations like AFM-Téléthon, which founded Genethon, this creates a profound ethical dilemma. Families affected by ultra-rare diseases often have no available treatment options and struggle even to access clinical trials. Clinical development for ultra-rare diseases should be publicly reimbursed, as trial participation itself represents the only therapeutic pathway for these patients.

He stresses that expecting charities to shoulder full late-stage development costs is not viable. Government-level intervention and alternative funding models are needed to ensure equitable access.

5. Regulatory and Reimbursement Landscape

Q: How do regulators and payers shape the development pathway for gene therapies?

A: Regulatory agencies such as the EMA and FDA have demonstrated strong openness to innovation in the gene therapy space. Europe is not inherently more restrictive than the U.S.; in fact, several ATMPs reached European markets before being approved in the United States.

The more significant barrier is reimbursement. Given the high price of ATMPs, payers require rigorous evidence demonstrating superiority over standard of care, long-term functional benefit, and durable biological effect. Randomized, placebo-controlled trials, long-term follow-ups, and real-world data are essential components of the evidence package.

6. Partnership Strategy and Access to Patients

Q: How does Genethon approach partnerships and collaborations?

A: Genethon evaluates partnerships through a single primary lens: would a collaboration accelerate patient access? This reflects Genethon’s origins as a patient-driven research organization. When partnerships can shorten development timelines or enhance global reach, they are actively pursued. However, for ultra-rare diseases, commercial partnerships are often infeasible due to limited market potential.

7. Role of Artificial Intelligence in Gene Therapy

Q: How is AI transforming Genethon’s approach to research and development?

A: Artificial intelligence is playing an increasingly important role in Genethon’s R&D. The organization recently published work in Nature Communications demonstrating the ability to design novel AAV capsids with organ-specific tropism using AI-driven approaches.

AI is also being applied in manufacturing, where predictive models may eventually enable digital bioreactor concepts. In clinical research, Genethon is using AI to develop rare-disease patient data hubs with French hospitals, improving the understanding of disease trajectories and enabling more objective monitoring of therapeutic response.

8. Europe’s Competitive Position in Gene Therapy

Q: How competitive is Europe globally in gene therapy innovation?

A: Europe has longstanding roots in the field — including the world’s first successful gene therapy trial in Paris for the ‘Bubble Babies’ syndrome. Europe today benefits from a mature scientific ecosystem, industrial capabilities, and long-term government initiatives.

One of the most significant initiatives is ‘France 2030,’ which established five national bioclusters targeting key therapeutic areas. Genother [the gene therapy biocluster chaired by Revah] received €140 million in funding and brings together manufacturing platforms, clinical networks, and training infrastructures. Europe is increasingly attracting talent from the U.S., reflecting strong scientific momentum and greater financial stability.

9. Leadership Insights for Biotech Innovators

Q: What mindset and skills should biotech leaders develop?

A: Successful biotech leaders must be both scientifically visionary and operationally grounded. They should be deeply passionate about science yet avoid falling in love with their own hypotheses. Equally important is a thorough understanding of CMC, regulatory strategy, manufacturability, and patient pathways.

Leadership in gene therapy requires balancing bold innovation with practical execution — ensuring that great science can ultimately become a real, manufacturable, reimbursable therapy for patients.