Nihal Sinha is Principal at F-Prime Capital and has worked with several advanced therapy companies including UK-based Orchard Therapeutics. Here Sinha talks about the unique relationship between F-Prime and Orchard Therapeutics, the commercialisation challenges the sector faces and the importance of working closely with academics.

An academic physician with a specialist research background in cognitive neuroscience, Sinha was a panel member at the Advanced Therapies Network (ATN) event on 14 May 2019, joining several other investors that are working in the advanced therapies sector.

F-Prime and Orchard Therapeutics

F-Prime has its main headquarters in Boston and a European office in London. Backed by FMR LLC, the parent company of Fidelity Investments, the firm has a global reach with multiple investments in the US, Europe and Asia. Within therapeutics F-Prime operates not only as a venture capitalist but also as a creator of companies. Many of those companies are in advanced therapies, including UK company Orchard Therapeutics. Orchard is a university spinout which F-Prime helped create in collaboration with Bobby Gaspar and Adrian Thrasher from University College London and Great Ormond Street Hospital, alongside scientists and clinicians from University of Manchester, Boston Children’s Hospital and University of California Los Angeles.

“We made a significant investment in the company,” says Sinha. “And we also invested a lot of our resources to help set up the company and recruit the team. Alex Pasteur (Partner at F-Prime Capital) was founding CEO for the first year of the company, Ben Auspitz (Partner at F-Prime’s Boston office) was founding Chairman and I also worked at the company.  It was essentially our primary focus in London for about a year.  Orchard now has offices in London, Boston and California and employs over 150 people.”

According to Sinha there were several factors that made Orchard Therapeutics stand out as a rare and potentially valuable opportunity for F-Prime. “Firstly, the company had a unique group of scientific founders from institutions with leading expertise in ex vivo gene therapy. Secondly, the clinical data behind the therapy was compelling and, at the time when we were considering investment, it had already treated patients with ADA-SCID (adenosine deaminase severe combined immunodeficiency) with 100 % survival rate.”

Another important factor for F-Prime was the multiple pipeline opportunities which could leverage the same technology. “The scientific founders demonstrated that the platform technology could be used to aggregate programmes across multiple rare diseases of high need and therefore build something of significant commercial scale.”

Recipe for an advanced therapies unicorn

Sinha was on the panel at ATN event on 14 May 2019 which discussed the factors that aid success in this field and the unique relationship that investors build with advanced therapies companies.

When talking about what aspects contribute to an advanced therapies company reaching unicorn status, Sinha prefers rather to talk about the qualities that F-Prime identified in Orchard Therapeutics and what he feels should be a basic premise across all life sciences companies. “When you think about what creates significant value in a therapeutics company, it’s ultimately in having products that will be used to change patients’ lives,” he says. “I think there are some attributes in Advanced Therapies Medicinal Products (ATMPs) that optimise the chances of this and a pivotal one is great science. The most experienced people in this field are mainly academics who have been working in this area for 20 years. I believe what we did right at Orchard was to form an incredibly intimate collaboration with the academics. The company was built around a group of phenomenal scientific founders who were deeply engaged and happy to transfer their knowledge and insight to the company.” Sinha also emphasised the importance of attracting the right team and Orchard has succeeded because it has attracted a great management team with significant experience in rare disease and gene therapy.

The attractiveness of investing in advanced therapies is often attributed to the potential of cell and gene therapy to transform people’s lives and provide cures. As such Sinha also believes that this impact must be clear and demonstrable.

“As an investor it’s only worth embracing the complexity of the manufacturing of advanced therapies if they have transformative potential,” says Sinha. “As such the therapy needs to target patients for whom this impact can be shown. What’s so amazing about this class of therapy is that because it often focusses on rare diseases, it’s possible to take something through a clinical development path relatively rapidly and nimbly to demonstrate the potential for significant impact.”

Commercialisation Challenges

Bringing together collective knowledge on how to overcome challenges to commercialisation is one of the aims of the Advanced Therapies Network (ATN). Having worked with Orchard Therapeutics and several other advanced therapies companies such as Adaptimmune from the UK as well as Caribou and Sana Biotechnology from the US, Sinha has a deep understanding of the commercialisation challenges that come with the territory of this new but fast-growing sector. He also has a sense of the ecosystem that is developing around these therapies to overcome these challenges.

“There are a lot of clever people that are working on methods, techniques and components that can help address the issues the sector faces, especially in manufacturing,” he says. “As academia begins to focus on these supportive approaches an industry is emerging and people are starting to invest in these companies. Examples include potent promoters in adeno associated viruses (AAVs) that potentially allow lower doses to be used, stable cell lines for lentivirus production to reduce costs and automated cell processing machines to create personalised medicines at the bedside. There is an ecosystem emerging around advanced therapies and, although there is no panacea to overcome the commercialisation challenges, we are getting better at identifying challenges and developing solutions.”

London and Advanced Therapies

F-Prime has its headquarters in the US and its European office is in London which is home to some of the world’s most prestigious academics who are working in advanced therapies. There are over 130 advanced therapies companies in the UK, with 60% residing in the greater south east of England.

“It is a tremendously exciting time for London in the advanced therapies sector,” says Sinha. “It’s really driven by the fact that the academic institutes here have been important centres for these sorts of therapies and have given rise to great scientific people who have now been able to enter into biotech companies which have been well funded. We have a wealth of relevant experience here and are developing an excellent ecosystem to make London a global leader in the sector.

“My work provides a fantastic combination of science, medicine, business and strategy, bringing together a lot of my interests. I love working with bright motivated entrepreneurs and scientists who are trying to make a positive impact on medicine and I’m looking forward to continuing to work in the advanced therapies sector which holds so much potential.”

Nihal Sinha was on the panel at the Advanced Therapies Network event on 14 May at Silicon Valley Bank.