Spur Therapeutics: one-time gene therapies to free patients from lifelong treatment

Problem to be solved

Inherited genetic disorders such as haemophilia and lysosomal storage disorders can be debilitating, life-threatening and costly to manage, often requiring regular injections or long-term supportive care. Many patients face lifelong disease burden and complications. There is a need for treatments that address the underlying cause and provide durable benefit from a single intervention.

Solution

Founded in 2015 based on work by Professor Amit Nathwani (UCL) in collaboration with St. Jude Children’s Research Hospital, Freeline Therapeutics - now Spur Therapeutics - developed liver-directed gene therapies using adeno-associated virus (AAV) vectors to deliver functional copies of missing or mutated genes. 

After early clinical work demonstrated stable expression of Factor IX from a single dose in haemophilia B, the team redesigned the gene carrier using a new synthetic capsid to improve gene transfer in human liver cells-supporting a ‘plug-and-play’ approach for additional genetic disorders.

How UCL Ventures helped

UCL Ventures supported the formation of Freeline alongside Syncona, helping to structure the company and licence multiple UCL gene therapy programmes to create a scalable pipeline. UCL Ventures also supported the underlying IP protection and the early commercial pathway, helping position the company to attract investment. This included participation from the UCL Technology Fund (UCLTF) alongside other investors as the company grew. As Redesigning the gene carrier explains: “The study showed that we could get stable expression of [blood clotting] Factor IX from a single dose. That was really exciting, because it was the first time anyone had shown that.”

Where is Spur Therapeutics now?

Freeline secured a £25m Series A investment in 2016, dosed the first patient in its haemophilia B programme in 2018, and progressed additional programmes including Fabry disease into the clinic in 2019. 

In 2020, the company completed an IPO on Nasdaq, raising $158.8m, and built manufacturing capability alongside its R&D laboratories in Stevenage to support rapid progression of multiple programmes. Next, the focus is on advancing late-stage development, expanding the pipeline and working towards broad patient access for one-time genetic medicines.

In 2024, Freeline was acquired by Syncona and merged with SwanBio to form Spur.