Expert’s Insights: Starting a CGT Biotech

With Thomas Wirth

Starting your own business is a challenge, but creating a Biotech comes with its own challenges. Recently, we spoke with Thomas Wirth, the Founder/Co-Founder of two Biotechnology businesses, Aurealis Therapeutics and Celloryx AG.

Founders must keep four things in mind if they want to be successful:

1. Be tenacious, but not stubborn. Listen to others and accept that you will have to make sacrifices along the way.
2. Stay true to yourself, don’t compromise on your values.
3. Choose your team carefully; it’s all about trust.
4. Make decisions based on scientific and business rationale, never on emotions.

In CGT development you have to be willing to accept compromises, whilst keeping the risk/benefit ratio in mind. Accept that not everything that’s desirable is also feasible. You have to keep the balance.

First Factors to Successfully Establishing a Biotech

There are two main ways a founder comes to start up a new Biotech business.

The first is an Academic who has specific research and data, and uses that data to create a company.

Thomas is from the other path. While he had Academic background knowledge, his skills were developed in a hands-on working environment. While leading a pre-clinical team at a Gene Therapy company, Thomas witnessed both the benefits and the bottlenecks of gene therapy-based medicines, and contemplated how to solve them.

When creating his business, Thomas realised that trying to control everything isn’t cohesive to success. He stated, “owning 10% of something is better than 100% of nothing. It’s all about how much a Founder is willing to give up to achieve a goal.” That isn’t to say you should compromise your values. Staying true to your values is essential to good business.

The Biggest Issues that Biotech Founders Experience

The first main issue is not knowing where to start, and this is for three reasons:

• You don’t have the right people at university telling you what you should be looking at.
• You don’t have the network to know who you could ask.
• You don’t have the funding to ask consultants.

When it comes to Cell and Gene Therapy, it’s an extremely fast-paced industry. Thomas’ expectation is that the life cycle of oncological gene therapy products is typically relatively short; meanwhile, development times are quite long.

While having experts is ideal, you also need to be realistic. For instance, doing everything per regulatory guidelines is the ideal. In reality, each CGT product is so unique that it has to be evaluated on a case-by-case basis.

Sometimes, it is not possible for you or your Biotech to accomplish a specific task. In this case, don’t be afraid to discuss with regulatory authorities. If you feel something is not viable, justify your view and propose solutions.

On the other hand, there is also a business aspect to be aware of. Doing anything and everything is probably every scientist’s dream; unfortunately, it’s unlikely to bring success since it dramatically increases your development timeline.

“No institutional VC will invest in your business if it takes five years to reach IND.”

Finding the right balance is crucial. Bringing the product to market as fast as possible is great for the business, but minimising risks and errors ensures it’s safe for the patient.

On the business side, VC firms typically want to understand the following:

• What is the value of your product?
• Is there a market for it?
• What is the patient population?
• Does the product have the proper protection?
• Will the protection last long enough to ensure ROI?

As current gene therapy products typically fall under the Orphan Drug Act/Orphan Designation, they receive market exclusivity for either 7(+2) years in the US or 10(+2) years in the EU. With this in mind, you could disagree with the last bullet point. However, Thomas argues that one should not rely on these incentives.

For example, in the Orphan Drug Act, there are three exemptions under which the USFDA can approve a product for which indication market exclusivity already exists. These are:

1. If the holder of the orphan drug exclusivity consents,
2. If the availability of sufficient quantities of the drug cannot be ensured by the ODA holder,
   or most importantly,
3. If the new drug is clinically superior to the already approved or licensed drug.

In the future, we’ll see more gene therapies developed for indications not falling under the ODA or Orphan Designation. With this in mind, one of the most common mistakes becomes more apparent.

When to file your IP

Filing it as soon as possible can be a good idea, since this protects your idea from the offset. This can be useful if there are several competitors with similar ideas.

On the other hand, it significantly reduces how long your product is protected on the market. The time to market for gene therapy products is much longer than for small molecules or antibodies.

The third problem people (particularly academics) face is publishing information far too soon. Their decision comes from a good place — wanting to raise awareness (and funding) for their business and product.

Unfortunately, doing this unnecessarily increases the risk to your business at such an early stage. A better option is to remain in stealth mode and postpone patenting for as long as possible.

Types of Experts You Need at the Beginning

Specialist knowledge is essential to running a successful business. Hiring and trusting people who fill the gaps in your knowledge is critical to developing long-term, consistent success.

“Have the right people before you establish anything”.

As a Biotech Founder, you should have the deepest understanding of the product. If you don’t want an Executive Role, make sure you’re in an Advisory Role.

You need three types of people to help you be successful. They are a:

1. CMO, who understands your product and how to make it.
2. Regulatory Expert, to ensure you have everything in place for filing an IND.
3. Business Specialist, to tell you what’s possible with your current resources.

Most importantly, you need someone capable of overseeing the entire project. They’ll be in charge of keeping the idea alive.

How to Find The Right Talent

When adding the ideal employee to your team, you don’t always want someone with the best knowledge and skills. Top leaders, like Thomas, believe that trust and culture fit is far more important.

“I always put trust in front of potential expertise, because you can train people.”

For instance, if a potential candidate lacks the skills, there’s always the option for them to learn and improve. If they don’t fit your company culture though, then they may end up damaging your business.

A poor culture fit could leave your company during a difficult time or reduce the morale of your existing team.

To avoid this, choosing your team based on who is the best overall fit is essential, rather than who has the best skills and experience.

Want to learn more about hiring in Biotech businesses? Read Biotech Vs Biopharma now!

Making a Move into Cell and Gene Therapy

Thomas’ greatest piece of advice for people looking to move into CGT is “stop being black and white”. It’s better to gain experience from a range of roles, and properly develop your understanding of how the business works.

It’s okay to challenge and re-interpret everything. For instance, when developing immune-oncology products, you may want to use mouse cytokines. Unsurprisingly, human cytokines may respond quite differently to mouse cytokines.

The question becomes ‘what is the value of information gained from mice’?

On the other hand, what would be the value of information gained from mice using human cytokines? And yet you must find a way to demonstrate that:

1. The drug is safe,
2. The drug is efficacious, and
3. The drug has an acceptable risk/benefit ratio.

The ability to challenge and re-interpret requirements needs excellent scientific and regulatory understanding to avoid roadblocks in your drug development path. If something isn’t feasible, bring it up, justify why not, and present potential solutions.

Be focused but open-minded. Work as a team. Have someone capable of overseeing the entire project, not only from a scientific aspect, but also from a business perspective.

Above everything, you must have fun. Don’t be compulsive; you won’t achieve your goal by force.

Thomas Wirth is a visionary and the Founder of two Biotechnology companies. With decades of experience in Cell and Gene Therapy businesses, Thomas has developed impeccable insights and understanding into this field. He is always willing to discuss his thoughts and findings; connect with Thomas to learn more.