Royal Prince Alfred Hospital

Hemgenix Gene Therapy Shows Sustained Efficacy in Hemophilia B Patients After Four Years

4 months ago

• Data from the HOPE-B trial demonstrates that Hemgenix (etranacogene dezaparvovec-drlb) provides long-term bleed protection for adults with hemophilia B. • Patients treated with Hemgenix experienced a 90% reduction in adjusted annualized bleeding rate (ABR) at year four compared to the lead-in period. • 94% of patients remained free of continuous prophylaxis treatment at year four, indicating sustained independence from regular infusions. • The gene therapy maintained a favorable safety profile, with most adverse events occurring within the first six months post-treatment.

Imugene Doses First Australian Patient in Phase 1b Trial of Azer-Cel for B-Cell Lymphoma

5 months ago

• Imugene has dosed the first Australian patient in a Phase 1b trial of azer-cel, an allogeneic CAR T-cell therapy, for relapsed or refractory diffuse large B-cell lymphoma (DLBCL). • The trial aims to evaluate azer-cel's tolerability, safety, and clinical activity in DLBCL patients who have failed previous autologous CAR T-cell treatments. • Azer-cel offers an off-the-shelf solution, potentially reducing treatment times and increasing patient access compared to traditional autologous CAR T-cell therapies. • Promising outcomes from US trial sites showed complete responses in subjects who received lymphodepletion chemotherapy and interleukin-2 (IL-2) after failing prior treatments.

Imugene's Azer-Cel Trial Opens First Australian Site; Neuren's Daybue Sales Exceed Expectations

6 months ago

• Imugene has initiated its Phase 1b clinical trial for azer-cel in Australia, marking a key advancement in its allogeneic CAR T-cell therapy development. • Neuren Pharmaceuticals reports Daybue sales surpassing US$250 million, triggering a US$50 million milestone payment and raising full-year income expectations. • Sigma Healthcare's shares surge following ACCC approval of its merger with Chemist Warehouse, poised to create a leading healthcare company. • Smartgroup Corporation sees its share price increase after Bell Potter reaffirmed a 'buy' rating with an improved price target of $10.00.

Early Palliative Care Integration Improves Outcomes in Hepatocellular Carcinoma Patients

7 months ago

• A pragmatic cluster-based randomized controlled trial is evaluating the impact of early palliative care referral on hepatocellular carcinoma (HCC) patients. • The study involves four Australian tertiary ambulatory HCC services, aiming to demonstrate the superiority of early palliative care over standard care. • The primary outcome is the improvement of palliative care symptoms, measured by a reduction in the liver-specific Edmonton Symptom Assessment Scale (ESAS) score. • The trial also assesses secondary outcomes such as quality of life, mortality, hospital admissions, and cost-effectiveness over a 5-year follow-up period.

Gene Therapy Breakthrough Effectively Cures 'Bubble Boy' Disease in Infants

6 years ago

• A groundbreaking gene therapy developed by US scientists has effectively cured infants with X-linked severe combined immunodeficiency (X-SCID), a rare and fatal genetic disorder that leaves children without functioning immune systems. • The treatment involves collecting patients' bone marrow, inserting a corrected copy of the mutated gene into their blood stem cells using a specially engineered virus, and returning the cells to the body after low-dose chemotherapy. • Within three months of treatment, seven of eight infants developed normal levels of multiple immune cell types, allowing them to fight off infections and lead normal lives without the need for ongoing treatment or protective isolation.