Discovery

Rare Diseases

OPK-546 Retinitis Pigmentosa

Rare Diseases

MTC-1203 Parkinson's Disease

Rare Diseases

MTC-1203 Huntington's Disease

Rare Diseases

TGF-β Orphan Bone Disease

Oncology

ProTriTAC Various (oncology)

Oncology

TriTAC Various (oncology)

Gene Therapy

LB-301 Crigler-Najjar

Gene Therapy

LB-201 A1ATD

Gene Therapy

LB-101 Hemophilia B

Pre-clinical

Oncology

AU-014 Gene Therapy

Oncology

AU-013 Immuno-Oncology

Oncology

AU-012 Primary bladder carcinoma

Oncology

AU-011 Cancers of the ocular surface

Oncology

AU-011 Choroidal metastases

Oncology

Target 3 and 4 (nucleases)

Oncology

Target 2 (Helicases )

Oncology

PolƟ (Polymerase theta)

Anti-inflammatory

LOX-topical Scarring

Anti-Infectives

SSAO/MPO Inflammation

Respiratory

RPL554 (MDI) COPD - Maintenance

Oncology

HPN328 Small Cell Lung Cancer (DLL3)

Oncology

HPN536 Ovarian cancer and other solid tumours (MSLN)

Oncology

HPN217 Multiple Myeloma (BCMA)

Gene Therapy

LB-001 Methylmalonic acidemia (MMA)

Oncology

AUTO6 NG Neuroblastoma; Melanoma; Osteosarcoma; SCLC

Oncology

AUTO7 Prostate Cancer

Oncology

AUTO5 TRBC2+ Peripheral TCL

Oncology

AUTO2 NG Multiple Myeloma

Oncology

AUTO3 NG B-Cell Malignancies

Phase 1

Respiratory

RL554 (nebulizer) Severe asthma

Oncology

VLS 101 Haematological Cancers

Anti-inflammatory

LOX-oral Cancer

Anti-inflammatory

LOXL-2 NASH/fibrosis

Oncology

HPN424 Prostate Cancer (PSMA)

Oncology

AUTO6 Neuroblastoma

Oncology

AUTO4 TRBC1+ Peripheral TCL

Oncology

AUTO2 Multiple Myeloma

Oncology

AUTO3 DLBCL

Oncology

AUTO3 Pediatric ALL

Oncology

AUTO1 Adult ALL

Oncology

AUTO1 Pediatric ALL

Phase 2

Anti-inflammatory

SSAO (PXS-4728A) Diabetic retinopathy

Anti-inflammatory

SSAO (PXS-4728A) NASH

Rare Diseases

IMR-687 Sickle Cell Disease

Anti-Infectives

APX001 Invasive Candidiasis

Oncology

AU-011 Ocular melanoma

Respiratory

RPL554 (DPI) COPD - Maintenance

Respiratory

RPL554 (nebulizer) COPD-Acute

Respiratory

RPL554 (nebulizer) Cystic Fibrosis

Respiratory

RPL554 (nebulizer) COPD maintenance

Anti-Infectives

REAKT Acute Kidney Injury

Phase 3

Anti-Infectives

SURE 2 Complicated Urinary Tract Infections

Anti-Infectives

SURE 3 Complicated intra-abdominal infections (cIAI)

Anti-Infectives

SURE 1 Uncomplicated Urinary Tract Infections

Anti-Infectives

ACCUTE Necrotising Soft Tissue Infection (NSTI)