Our expertise & pipeline

A potential first-in-class, investigational G-Quadruplex (G4)-selective transcription inhibitor designed for advanced or metastatic solid tumours. Currently being trialled in pancreatic cancer.

Pancreatic cancer is more common in older people: more than 45% of people diagnosed are 75 and over. In England, pancreatic cancer is more common in people living in more deprived areas.

It is more common in White and Black people than in Asian people and in people of mixed or multiple ethnicity.

A gene therapy to restore cone function via subretinal injection to cone receptors in the back of the eye. It was designed with a synthetic promoter associated with strong gene expression, to account for the larger amount of protein needed to restore cone function in achromatopsia (ACHM) patients with a CNGA3 gene mutation (~1 in 30,000 and 30-40% cases).

ACHM causes complete colour blindness, extreme light sensitivity, involuntary eye movements (nystagmus), and severely reduced visual acuity.

A gene therapy to restore cone function via subretinal injection to cone receptors in the back of the eye. It is indicated in adults and children with achromatopsia caused by mutations in the CNGB3 gene (~1 in 30,000; 40-50% of cases).

ACHM causes complete colour blindness, extreme light sensitivity, involuntary eye movements (nystagmus), and severely reduced visual acuity.

A gene therapy in which the expression of a codon-optimised RPE65 gene is driven by a novel synthetic retinal pigment epithelium (a thin layer of cells at the back of the eye) cell-specific promoter.

The RPE65 protein is essential for rod function because it recycles the light sensing machinery in rod photoreceptors. A deficiency of RPE65 results in Leber congenital amaurosis (LCA), which occurs in 2 to 3 per 100,000 newborns. It is one of the most common causes of blindness in children.

CAR T-cell therapy targeting CD19 and CD22 expressing cells for paediatric Acute Lymphocytic Leukaemia (ALL), the most common form of childhood leukaemia and accounts for around 30% of all paediatric cancers.

In ALL, the bone marrow makes too many lymphocytes (a type of white blood cell).

CAR T-cell therapy targeting cells expressing the T-cell receptor β-chain constant domain 1 (TRBC1). It is indicated for peripheral T-cell lymphoma (PTCL), a subtype of non-Hodgkin’s lymphoma - defined as a diverse group of aggressive lymphomas that develop from mature-stage white blood cells called T-cells and natural killer (NK) cells.

PTCL is <1 case per 100,000 people in the United States.

A CAR T-cell therapy targeting cells expressing CD19 and B-cell maturation antigen (BCMA) for multiple myeloma. This bone marrow cancer often affects several areas of the body, such as the spine, skull, pelvis and ribs.

The incidence of multiple myeloma is 4.5-6.0 per 100,000 people per year and the mortality rate is 4.1 per 100,000.

β-Glucocerebrosidase (GBA) AAV gene therapy for the treatment of Gauchers disease (or GBA deficiency).

Gauchers affects 1 in 40,000 people, but in the Ashkenazi Jewish population, it affects 1 in 450 people.

A first-in-class bispecific T-cell engager, which targets ROR1 tumours, killing cancer cells without depleting B cells. Initially focused on haematological malignancies such as Chronic Lymphocytic Leukaemia (CLL) to establish proof of concept prior to moving to hard-to-treat solid tumours.

CLL is the most common adult leukaemia (~38% of cases).

A lentiviral ex vivo autologous gene therapy for mucopolysaccharidosis type I (MPS-I) or Hurler syndrome. An enzyme deficiency (alpha-L-iduronidase (IDUA) lysosomal enzyme) leads to a build-up of sugar molecules (glycosaminoglycans or GAGs )across across organs. This causes systems including neurocognitive impairment, skeletal deformity, loss of vision and hearing, and cardiovascular and pulmonary complications. It affects 1 in 25,000.

Cell-based treatment for Wet Age-Related Macular Degeneration (Wet AMD). This is blurred vision or a blind spot in the central vision, usually caused by blood vessels that leak fluid or blood into the macula. Retinal pigment epithelium (RPE) cells die leading to the blind spot, but this treatment re-introduces healthy RPE.

Affects c.165 million people globally.

Oral formulation of Ornithine Phenyl Acetate (OP) for the treatment of Hepatic Encephalopathy (HE). HE occurs when a patient is in liver failure and there is an ammonia build-up in the brain which leads to brain swelling, cognitive impairment and death. OP is an ammonia lowering agent. 62% of hepatic cirrhosis patients have HE.

TLR4 antagonist for the treatment of acute liver failure, which reduces inflammation. Fewer than 10 cases per 1,000,000 in the developed world.

An AAV-based gene therapy to treat the most common form of X-linked retinitis pigmentosa (XLRP), an incurable form of genetic blindness in men (~1 in 15,000). Caused by mutations in RPGR ORF15 gene; both rods and cones photoreceptors require RPGR ORF15 to function.

Adenosine deaminase (ADA) deficiency is an inherited disorder causing severe combined immunodeficiency (SCID). Patients have virtually no immune protection against bacteria, viruses and fungi.

It affects 1 in 1,000,000 newborn children.

IV formulation of Ornithine Phenyl Acetate (OP) for the treatment of Hepatic Encephalopathy (HE). HE occurs when a patient is in liver failure and there is an ammonia build-up in the brain which leads to brain swelling, cognitive impairment and death. OP is an ammonia lowering agent.

There are currently 62,000 cases per 100,000 patients with hepatic cirrhosis.

Ridinilazole (previously known as SMT19969) is an investigational small molecule antibiotic for oral administration to treat Clostridioides difficile infection (CDI). CDI is a hospital-acquired infection and occurs in 8.3 patients per 10,000 admissions.

A CAR T-cell therapy using CD19 as the target. It is indicated for the treatment of adult Acute Lymphocytic Leukaemia (ALL), a type of cancer in which the bone marrow makes too many lymphocytes (a type of white blood cell).

The estimated worldwide annual incidence of adult ALL is about one in 100,000.

Kogenate Bayer is used for the treatment and prevention of bleeding in patients of all ages with haemophilia A (an inherited bleeding disorder caused by lack of factor VIII, a clotting protein). It contains the active substance octocog alfa (human coagulation factor VIII).

An oncolytic herpes simplex virus (HSV-1) expressing genetically-modifiedCSF for the treatment of various tumours. It is designed to selectively replicate inside cancer cells and destroy them, while simultaneously stimulating the patient's immune system to attack other tumours.

It is sold by Biovex / Amgen.

Basiliximab (trade name Simulect) is a chimeric mouse-human monoclonal antibody to the α chain (CD25) of the IL-2 receptor of T-cells. It is used to prevent rejection in organ transplantation, especially in kidney transplants. It is sold by Novartis.

Used to treat narcolepsy in adults, adolescents and children from six years of age by increasing histamine levels in the brain.

Narcolepsy is a long-term sleep disorder, which affects the brain’s ability to regulate the normal sleep-wake cycle. This leads to symptoms such as an irresistible urge to sleep, even at inappropriate times and places, and disturbed night-time sleep. Some patients also have episodes of severe muscle weakness (cataplexy) that can cause collapse.