Across our portfolio we now have 26 programmes in the clinic, focusing on areas of high unmet medical need

Key

Respiratory

Anti-infectives

Immunology & inflammation

Rare diseases

Oncology

Discovery

Very initial stages of testing and validating scientific discoveries.

LogicBio

LB-101 Hae mophilia B

LogicBio

LB-201 A1ATD (liver disease)

LogicBio

LB-301 Crigler-Najjar

OptiKira

OPK-546 Retinitis Pigmentosa

Mitoconix

MTC-1203 Huntington's Disease

Mitoconix

MTC-1203 Parkinson's Disease

Pharmaxis

SSAO/MPO Inflammation

Pharmaxis

LOX (topical) Scarring

PreciThera

TGF-θ Orphan Bone Disease

Pre-clinical

At this, the focus is on researching the feasibility and safety of a treatment before commencing clinical trials.

Artios

Pol Theta (Polθ)

Artios

Target 2 (Helicase)

Artios

TARGET 3 and 4 (Nucleases)

Autolus

AUTO7 Prostate Cancer

Autolus

AUTO2 NG Multiple Myeloma

Autolus

AUTO3 NG B-Cell Malignancies

Autolus

AUTO6 NG Neuroblastoma; Melanoma; Osteosarcoma; SCLC (GD2)

Autolus

AUTO5 T-Cell Lymphoma (TRBC2)

Harpoon

HPN536 Varios Solid Tumours (TriTAC) - Mesothelin

Harpoon

HPN217 Multiple Myeloma BCMA (TriTAC)

Harpoon

HPN823 Small Cell Lung Cancer (TriTAC)

Harpoon

Various - oncology (TriTAC)

Harpoon

Various - oncology (ProTriTAC)

LogicBio

LB-001 methylmalonic acidemia (MMA)

Verona

COPD Maintenance (MDI)

Phase One

This is the first time a product is tested in humans. The focus at this stage is testing the side effects and safety.

Autolus

AUTO2 Multiple myeloma (BCMA and TACI)

Autolus

AUTO3 DLBCL (CD19 and CD22)

Autolus

AUTO3 pALL (CD19 & CD22)

Autolus

AUTO6 Neuroblastoma (GD2)

Autolus

AUTO1 aALL (CD19)

Autolus

AUTO1 pALL (CD19)

Autolus

AUTO1 pALL (CD19)

Pharmaxis

SSAO Diabetic retinopathy

Pharmaxis

SSAO NASH

Harpoon

PN424 Prostate Cancer

Pharmaxis

LOXL-2 NASH, Fibrosis

Autolus

AUTO4 T-cell lymphoma (TRBC1)

Verona

Severe Asthma (Nebulizer)

VelosBio

VLS 101 (Haematological Cancers)

Pharmaxis

LOX (oral) cancer

Phase Two

Phase 2 involves further trials testing the efficacy and safety and different dosing levels.

Atox Bio

REAKT AKI

Verona

COPD Acute (Nebulizer)

Verona

COPD Fibrosis (Nebulizer)

Verona

COPD Maintenance (Nebulizer)

Amplyx

APX001 Invasive fungal infections

Verona

COPD Maintenance (DPI)

Aura

AU-011 Ocular Melanoma

Phase Three/Pivotal

This is the final stage of testing before registration. Phase 3 trials focus on testing the effectiveness of the new product compared to existing treatments or to a placebo.

Atox Bio

ACCUTE NSTI

Iterum

Complicated UTI

Iterum

Uncomplicated UTI

Iterum

Complicated intra abdominal infections