Advanced Therapies event highlights UCLB’s world-leading work

This week’s Advanced Therapies conference at ExCel provided a chance for UCLB to collaborate with UCL’s Translational Research Office to promote our commercialisation successes as well as looking to the future possibilities, says UCLB Director of Biopharm Richard Fagan. 

“UCLB’s advanced therapies development pipeline is akin to that of a large-sized pharmaceutical company. It includes five therapies in Phase III clinical trials, offering exciting potential treatments for conditions including forms of retinal disease and leukaemia.” 

“This week’s presence of the UCL TRO at the Advanced Therapies Congress at ExCel, London, gave us a chance to highlight UCLB’s recent innovation in the field of advanced therapies” 

Autolus: Revolutionising cancer treatment 

Autolus Therapeutics made significant strides in the field of cancer treatment recently. Its CAR-T cell therapy, obecabtagene autoleucel (obe-cel), has shown promising results in treating adults with relapsed/refractory B-cell acute lymphoblastic leukemia (r/r B-ALL). This innovative therapy reprograms a patient’s immune cells to target and destroy cancer cells, offering a new lease on life for those battling this aggressive blood cancer.  

The FELIX trial, led by researchers at UCL and UCLH, demonstrated the efficacy of obe-cel in reducing immune toxicity and improving patient outcomes. Autolus’s success is a testament to the collaborative efforts between UCLB and the TRO, which have been instrumental in protecting and licensing obe-cel to Autolus in 2018 enabling them to raise further capital to develop it to the market and ultimately patient benefit.  

In November 2024, Autolus gained FDA approval for their CAR-T cell therapy, marketed as AUCATZYL®. 

MeiraGTx: Advancing diverse gene therapy solutions 

MeiraGTx is pioneering gene therapy treatments for inherited eye diseases. Its therapy for X-linked retinitis pigmentosa (XLRP), botaretigene sparoparvovec, a condition that leads to blindness, is currently in late Phase 3 clinical trials, based on the research of Professor Robin Ali at the UCL Institute of Ophthalmology.  

And, just last week, MeiraGTx announced it has entered a strategic collaboration with Hologen, a spinout from UCL and KCL, created with the support of UCLB. The collaboration aims to accelerate development of AAV-GAD for Parkinson’s disease and industrialise MeiraGTx’s proprietary manufacturing process.  

EpilepsyGTx: On the cutting edge 

Focused on research and development of cutting-edge gene therapies to treat focal refractory epilepsy, EpilepsyGTx raised $10m in seed funding last year. The financing was led by the UCL Technology Fund, with participation from Health Technology Holding. 

The funds raised will be used to complete the preclinical studies for EpilepsyGTx’ lead gene therapy programme EPY201, and to prepare a groundbreaking first-in-human, Phase 1/2a clinical study with EPY201 in focal refractory epilepsy. 

EpilepsyGTx’s mission is to make patients with focal refractory epilepsy seizure-free. It is developing a portfolio of cutting-edge gene therapies which are based on pioneering research from the UCL Queen Square Institute of Neurology. 

Future gazing 

In the coming years, UCLB’s advanced therapies are poised for critical progress. Therapies currently in Phase III trials, such as MeiraGTx’s AAV-RPGR for XLRP hold great promise. Let’s hope next year’s Advanced Therapies brings with it more great news.