CSL Behring

Complement inhibitors market is growing due to the complement system's role in rare and chronic diseases. Over 40 companies are developing 50+ pipeline inhibitors, including Gefurulimab, ANX005, DNTH103, and others. Recent milestones include Q32 Bio presenting ADX-097 data at ASN Kidney Week 2024 and ReAlta Life Sciences dosing the first patient in a Phase II study of RLS-0071.

AusBiotech 2024 will feature over 260 speakers and 60 sessions, addressing global biotech trends, research, and industry advancements. Key topics include artificial heart technology, quantum computing in biotech, and medical manufacturing. The event also highlights emerging biotechnologies in agriculture and cell and gene therapies, supported by government initiatives and strategic partnerships. Australia's biotech sector is rapidly advancing, driven by innovation, government support, and strategic geographical clustering near research institutions.

A single dose of CRISPR-based NTLA-2002 reduced angioedema attacks by 75-77% in patients with hereditary angioedema, with 80% of patients in the 50 mg group experiencing complete response.

Be Biopharma raised $82m to advance hemophilia B gene therapy BE-101 to Phase I/II trial, adding ex-Roche exec Suha Patel. BE-101, an FDA-approved gene therapy, aims to provide durable FIX replacement for hemophilia B patients.

Self-amplifying mRNA (samRNA) vaccines face scientific and regulatory challenges, but experts believe they will eventually enter the market, especially for infectious diseases and cancer. Despite initial setbacks, Gennova Biopharmaceuticals' Gemcovac and Arcturus Therapeutics' Kostaive have gained approvals in India and Japan, respectively. The success of Kostaive is attributed to strategic timing and unique trial conditions. While samRNA therapeutics offer potential benefits over standard mRNA vaccines, technical hurdles and safety concerns remain, affecting their widespread adoption.

The hemophilia drugs market focuses on treatments for hemophilia A and B, including factor replacement, gene therapy, and bypassing agents. Key drivers are the increasing prevalence of hemophilia, advances in biotechnology, and heightened awareness of early diagnosis. Major companies include Grifols SA, CSL Behring, and Pfizer, Inc. The market is segmented by treatment type, product, route of administration, and geography, with North America and Europe leading due to advanced healthcare infrastructure and supportive regulatory frameworks. Asia-Pacific is expected to see rapid growth due to improving healthcare facilities and increasing awareness.

FDA approves Pfizer's Hympavzi, a new weekly injection for hemophilia A and B, which reduces TFPI activity to prevent bleeding. This expands Pfizer's hemophilia treatments, offering flexibility and lower bleeding rates compared to traditional factor replacement. Common side effects include injection site reactions, headache, and itching.

The immunoglobulin market, valued at US$ 13.36 billion in 2023, is projected to reach US$ 28.70 billion by 2034, driven by its use in treating immunodeficiency and other applications. Key growth factors include immunodeficiency disorders, infections, and thrombocytopenia. North America leads with a 46% share, while Asia-Pacific is expected to grow fastest due to increased health spending and prevalence of autoimmune diseases. Recent advancements include subcutaneous injection therapies and acquisitions by major pharmaceutical companies.

DPHARM 2024 focused on innovations in clinical trials, featuring keynotes on ROI, DCTs, AI, and patient perspectives. Panels discussed efficiency, AI use, and ecosystem approaches. Outside-industry speakers offered unique insights. Technologists showcased patient-centric services. Sponsors highlighted innovation and collaboration. DPHARM Idol Disrupt finalists presented disruptive technologies, with Power winning for its patient-matching platform. The event aimed to improve clinical trials for faster, more efficient medicine delivery.