Our portfolio

Syncona has a selective, focused portfolio of high quality healthcare businesses in truly innovative areas of science

We take significant ownership stakes companies that we can own at least to commercialisation, to work in deep partnership with those portfolio companies such that we can enhance their chances of success, and to target areas of dramatic efficacy.

Today, our portfolio is made up of a small group of healthcare companies with the potential to be global leaders in areas of high unmet need. In time, we are seeking to build a portfolio of no more than 15-20 companies which can deliver transformational treatments to patients. 

Pipeline

Key:

Established
Maturing
Developing
Portfolio company
Syncona focuses on delivering dramatic efficacy for patients in disease areas of high unmet need.
Disease area
Syncona seeks the best scientific ideas from world leading academic individuals and organisations. We work in partnership to found a scalable commercial business which can take its treatment all the way to market and to patients.
Best ideas
Before companies start clinical trials on a treatment, they conduct extensive pre-clinical studies, involving in vivo or in vitro experiments using wide-ranging doses of the product to obtain preliminary efficacy and safety information.
Pre-clinical development
Treatments under development are tested in humans for the first time, usually in a small group of healthy volunteers initially. As testing progresses, approval will be sought for a trial involving a larger group of people, often with patients who have the condition which the potential treatment is targeting. The objective of this phase is to determine any safety issues with the treatment and establish that it is effective at treating the disease.
Phase I/II
The final stage of the clinical process involving a larger trial of a group of people with the disease being targeted. The objective is to demonstrate the safety and effectiveness of the new treatment, confirm effective dosing levels and compare results against any currently achieved by existing treatments.
Phase III
The treatment has become a marketed product and is being used to treat patients with meaningful impact.
Transformational treatment
Blue Earth
Recurrent prostate cancer
Recurrent prostate cancer

Blue Earth Diagnostics was founded in March 2014 to develop and commercialise molecular imaging agents, addressing areas of high unmet medical need. The company’s first approved and commercially available product, Axumin®, is clinically proven to target and image prostate cancer cells. Axumin was reviewed by the US Food and Drug Administration and was granted US regulatory approval in Q2 2016. Marketing authorisation in Europe was granted by the European Medicines Agency in Q2 2017. Blue Earth Diagnostics is investigating the utility of Axumin, the imaging of other cancers, such as glioma. In May 2018, Blue Earth Diagnostics expanded its prostate cancer portfolio through the acquisition of exclusive, worldwide rights to a broad family of Prostate Specific Membrane Antigen (‘PSMA’)-targeted radiohybrid (‘rh’) agents for cancer. rhPSMA is a clinical-stage, investigational class of theranostic compounds, with potential applications in both the imaging and treatment of prostate cancer.

Nightstar
NSR-REP1 Choroideremia
NSR-REP1 Choroideremia

Nightstar is a leading clinical-stage gene therapy company focused on developing and commercialising novel one-time treatments for patients suffering from rare inherited retinal diseases that would otherwise progress to blindness. Nightstar’s lead product candidate, NSR-REP1, is currently in Phase 3 development for the treatment of patients with Choroideremia, a rare, degenerative, genetic retinal disorder that has no current treatments and affects approximately one in every 50,000 people. 

Positive results from a Phase 1/2 trial of NSR-REP1 were published in The Lancet in 2014 and in The New England Journal of Medicine in 2016. Nightstar’s second product candidate, NSR-RPGR, is currently being evaluated in a Phase 1/2 clinical trial for the treatment of patients with X-linked Retinitis Pigmentosa, an inherited X-linked recessive retinal disease that affects approximately one in every 40,000 people.

Nightstar
NSR-RPGR XLRP
NSR-RPGR XLRP

Nightstar is a leading clinical-stage gene therapy company focused on developing and commercialising novel one-time treatments for patients suffering from rare inherited retinal diseases that would otherwise progress to blindness. Nightstar’s lead product candidate, NSR-REP1, is currently in Phase 3 development for the treatment of patients with Choroideremia, a rare, degenerative, genetic retinal disorder that has no current treatments and affects approximately one in every 50,000 people. 

Positive results from a Phase 1/2 trial of NSR-REP1 were published in The Lancet in 2014 and in The New England Journal of Medicine in 2016. Nightstar’s second product candidate, NSR-RPGR, is currently being evaluated in a Phase 1/2 clinical trial for the treatment of patients with X-linked Retinitis Pigmentosa, an inherited X-linked recessive retinal disease that affects approximately one in every 40,000 people.

Autolus
AUTO2 Multiple Myeloma
AUTO2 Multiple Myeloma

Autolus is a clinical-stage biopharmaceutical company developing next-generation programmed T cell therapies for the treatment of cancer. 

Using its broad suite of proprietary and modular T cell programming technologies, Autolus is engineering precisely targeted, controlled and highly active T cell therapies that
are designed to better recognise cancer cells, break down their defence mechanisms and eliminate these cells.

Autolus has a pipeline of product candidates in development for the treatment of haematological malignancies and solid tumours.

Autolus believes its programmed T cell therapies have the potential to be best-in-class and offer cancer patients substantial benefits over the existing standard of care, including the potential for cure in some patients. 

Autolus
AUTO3 DLBCL
AUTO3 DLBCL

Autolus is a clinical-stage biopharmaceutical company developing next-generation programmed T cell therapies for the treatment of cancer. 

Using its broad suite of proprietary and modular T cell programming technologies, Autolus is engineering precisely targeted, controlled and highly active T cell therapies that
are designed to better recognise cancer cells, break down their defence mechanisms and eliminate these cells.

Autolus has a pipeline of product candidates in development for the treatment of haematological malignancies and solid tumours.

Autolus believes its programmed T cell therapies have the potential to be best-in-class and offer cancer patients substantial benefits over the existing standard of care, including the potential for cure in some patients. 

Autolus
AUTO3 pALL
AUTO3 pALL

Autolus is a clinical-stage biopharmaceutical company developing next-generation programmed T cell therapies for the treatment of cancer. 

Using its broad suite of proprietary and modular T cell programming technologies, Autolus is engineering precisely targeted, controlled and highly active T cell therapies that
are designed to better recognise cancer cells, break down their defence mechanisms and eliminate these cells.

Autolus has a pipeline of product candidates in development for the treatment of haematological malignancies and solid tumours.

Autolus believes its programmed T cell therapies have the potential to be best-in-class and offer cancer patients substantial benefits over the existing standard of care, including the potential for cure in some patients. 

Autolus (academic partners)
AUTO1 pALL
AUTO1 pALL

Autolus is a clinical-stage biopharmaceutical company developing next-generation programmed T cell therapies for the treatment of cancer. 

Using its broad suite of proprietary and modular T cell programming technologies, Autolus is engineering precisely targeted, controlled and highly active T cell therapies that
are designed to better recognise cancer cells, break down their defence mechanisms and eliminate these cells.

Autolus has a pipeline of product candidates in development for the treatment of haematological malignancies and solid tumours.

Autolus believes its programmed T cell therapies have the potential to be best-in-class and offer cancer patients substantial benefits over the existing standard of care, including the potential for cure in some patients. 

Autolus (academic partners)
AUTO6 Neuroblastoma
AUTO6 Neuroblastoma

Autolus is a clinical-stage biopharmaceutical company developing next-generation programmed T cell therapies for the treatment of cancer. 

Using its broad suite of proprietary and modular T cell programming technologies, Autolus is engineering precisely targeted, controlled and highly active T cell therapies that
are designed to better recognise cancer cells, break down their defence mechanisms and eliminate these cells.

Autolus has a pipeline of product candidates in development for the treatment of haematological malignancies and solid tumours.

Autolus believes its programmed T cell therapies have the potential to be best-in-class and offer cancer patients substantial benefits over the existing standard of care, including the potential for cure in some patients. 

Freeline
Haemophilia B
Haemophilia B

Freeline is a clinical-stage biotechnology company based in the UK and Germany. Freeline’s vision is to create better lives for people suffering from chronic systemic diseases using the potential of gene therapy as a one-off curative treatment. Freeline has a clear focus on AAV-based gene therapy targeting the liver with the aim to provide treatments for diseases with significant unmet needs. 

Freeline’s gene therapy treatment builds upon the pioneering work by the Freeline CSO, Professor Amit Nathwani, Professor of Haematology at UCL, which has already transformed the lives of patients by providing safe, effective and reliable gene therapy for people with Haemophilia B.

Freeline’s proprietary AAV capsid is optimised to effectively transduce human liver cells and thus provide sustainable, high levels of the desired protein. This strong capsid performance is being validated in the Haemophilia B patients and sets the stage for potentially curative products in a wide range of diseases which have not to date been treatable with one-time treatment.

Autolus (academic partners)
AUTO1 aALL
AUTO1 aALL

Autolus is a clinical-stage biopharmaceutical company developing next-generation programmed T cell therapies for the treatment of cancer. 

Using its broad suite of proprietary and modular T cell programming technologies, Autolus is engineering precisely targeted, controlled and highly active T cell therapies that
are designed to better recognise cancer cells, break down their defence mechanisms and eliminate these cells.

Autolus has a pipeline of product candidates in development for the treatment of haematological malignancies and solid tumours.

Autolus believes its programmed T cell therapies have the potential to be best-in-class and offer cancer patients substantial benefits over the existing standard of care, including the potential for cure in some patients. 

Autolus
AUTO4 T cell Lymphoma
AUTO4 T cell Lymphoma

Autolus is a clinical-stage biopharmaceutical company developing next-generation programmed T cell therapies for the treatment of cancer. 

Using its broad suite of proprietary and modular T cell programming technologies, Autolus is engineering precisely targeted, controlled and highly active T cell therapies that
are designed to better recognise cancer cells, break down their defence mechanisms and eliminate these cells.

Autolus has a pipeline of product candidates in development for the treatment of haematological malignancies and solid tumours.

Autolus believes its programmed T cell therapies have the potential to be best-in-class and offer cancer patients substantial benefits over the existing standard of care, including the potential for cure in some patients. 

Gyroscope
Dry AMD
Dry AMD

Gyroscope is committed to changing ophthalmology and fighting the devastating impact of sight loss. It is exploiting the convergence of breakthroughs in genetics and proprietary insights into the role of the complement system in AMD to develop a series of one-off gene therapy treatments for dry AMD and other chronic eye diseases.

The company was founded in 2016 with investments from Syncona and Cambridge Enterprise. Gyroscope is located in Stevenage at the Bioscience Catalyst.

Nightstar
Stargardt's disease
Stargardt's disease

Nightstar is a leading clinical-stage gene therapy company focused on developing and commercialising novel one-time treatments for patients suffering from rare inherited retinal diseases that would otherwise progress to blindness. Nightstar’s lead product candidate, NSR-REP1, is currently in Phase 3 development for the treatment of patients with Choroideremia, a rare, degenerative, genetic retinal disorder that has no current treatments and affects approximately one in every 50,000 people. 

Positive results from a Phase 1/2 trial of NSR-REP1 were published in The Lancet in 2014 and in The New England Journal of Medicine in 2016. Nightstar’s second product candidate, NSR-RPGR, is currently being evaluated in a Phase 1/2 clinical trial for the treatment of patients with X-linked Retinitis Pigmentosa, an inherited X-linked recessive retinal disease that affects approximately one in every 40,000 people.

Achilles
Non small cell lung cancer
Non small cell lung cancer

Achilles Therapeutics’ mission is to develop next-generation personalised therapies that harness the immune system to destroy cancer cells, with the potential to cure patients. These immunotherapies target clonal tumour neoantigens, protein markers unique to each individual that are expressed on the surface of every cancer cell. Achilles uses DNA sequencing data from each patient, together with its proprietary bioinformatics technology, to identify clonal neoantigens specific to that patient and to develop personalised therapies. 

Targeting multiple clonal mutations present in every tumour cell will reduce the risk that new mutations can induce immune evasion and therapeutic resistance, as occurs with many conventional approaches.

SwanBio
Neurodegenerative disorder
Neurodegenerative disorder

SwanBio Therapeutics is a gene therapy company focused on the development and commercialisation of genetically defined therapies for the treatment of neurological disorders. The company, which is located in Boston and Philadelphia, is building on its proprietary delivery and manufacturing know-how to enable it to deliver its products commercially, which it will leverage for each of its pipeline products.

The business is based on work by Florian Eichler, a founder of SwanBio and Associate Professor of Neurology at Harvard Medical School and Assistant in Neurology, Director of the Leukodystrophy Clinic, and Director of the Center for Rare Neurological Diseases at Massachusetts General Hospital. Its lead programme is in development for a monogenic neurodegenerative condition impacting approximately one in 17,000 people.

Multiple undisclosed pre-clinical programmes