HI-Bio: A Biogen Company’s Post

Yesterday was #RareDiseaseDay. The annual observance highlights how far the #biopharma industry has come and serves as a wake-up call for how far it must still go to meet the needs of #raredisease patients. Despite recent investment and breakthroughs for #rarediseases, experts told BioSpace that helping a much larger patient population will require cross-sector support and desperately needed funding. We looked at some of the investments to date, as well as the biopharma industry’s promising pipelines and what is required to bring more groundbreaking therapies to patients with unmet medical needs. #Patientadvocacy groups have been critical to the development of the first marketed drugs for certain rare diseases, including progeria and Friedreich's ataxia. We spoke with leaders of these groups formed by families coping with rare diseases, providing case studies on their partnerships with biopharma companies. https://lnkd.in/g7R6hU23 #RareDiseaseDay2024 #genetherapy #genetherapies #duchenne #duchennemusculardystrophy #biopharmaceuticals

For #RareDiseaseDay2024, here's a round-up of the latest breakthroughs in the treatment of rare disease from Drug Discovery World. #rarediseaseday #raredisease #drugdiscovery https://lnkd.in/exgtVJFz

Yesterday, the FDA announced plans to launch a Rare Disease Innovation Hub that will utilise new and collaborative approaches to expedite the development and approval of safe and effective drugs and biologics that treat rare diseases. One out of every 10 people in the US are affected by a rare disease and around half of these people are children. Many rare conditions are life-threatening, and most do not have approved treatments. The new Hub will work across rare diseases with a focus on products intended for smaller populations or for diseases where the natural history is variable and not fully understood. It will serve as a single point of connection and engagement with the rare disease community; enhance agency collaboration to address common scientific, clinical and policy issues related to rare disease product development; and advance regulatory science. Having supported organisations across the globe with the development of medicines for rare diseases, Boyds warmly welcomes this promising initiative which ultimately aims to improve outcomes for people living with rare diseases by providing new therapeutic options. Find out more: https://lnkd.in/egJKYwvs #rarediseases #fda #innovationhub #medicine

Vertex Pharmaceuticals to Acquire Alpine Immune Sciences for $4.9 Billion US-based Vertex Pharmaceuticals has agreed to pay approximately $4.9 billion in cash to acquire Alpine Immune Sciences, Inc., a compatriot #biotechnology company focused on discovering and developing innovative, protein-based #immunotherapies. The transaction was unanimously approved by the boards of directors of both companies and is expected to close later this quarter. Alpine’s lead molecule, povetacicept (ALPN-303), is a dual antagonist of BAFF (B cell activating factor) and APRIL (a proliferation inducing ligand). Through Phase 2 development, povetacicept has shown potential best-in-class efficacy in IgA nephropathy (IgAN), the companies said in a joint statement. IgAN is a serious, progressive, autoimmune disease of the kidney that can lead to end-stage-renal disease. So far, there are no approved therapies that target the underlying cause of IgAN. Povetacicept is on track to enter Phase 3 clinical development in the second half of 2024. Read more on #CMI online: https://lnkd.in/ebDN63QP

Big news!   Unveiling Calluna Pharma – a clinical stage company pioneering a breakthrough approach to treating inflammatory and fibrotic diseases by leveraging the body’s innate immune system. Calluna Pharma is the result of the successful merger between Arxx Therapeutics and Oxitope Pharma, backed by existing investors Forbion, Sarsia, p53, and Investinor.   Together, we are forging a path towards groundbreaking advancements in harnessing the innate immune system to revolutionize disease modulating therapies. Our combined expertise in the realm of innate immunity, positions Calluna as a leader in the field. Our robust pipeline of selective antibodies targeting innate immune amplifiers of disease, enables us to fulfil unmet medical needs across a spectrum of chronic and acute diseases.   Join us on this exciting journey as we develop novel therapies with the aim of reshaping the landscape of immunological diseases. Stay tuned for updates as we continue to grow and drive progress in the field.   Want to learn more in the meantime?   Visit our website here: https://lnkd.in/e37y2VKy   #PressRelease #Biologics #DrugDevelopment #Clinical #PrecisionMedicine  

In our latest article published in PLOS Neglected Tropical Diseases, we reviewed the snakebite envenoming (SBE) therapeutics pipeline and antivenom market. Here’s what we found: 💉 We identified 127 products, which may seem sizeable, but this does not translate into a robust antivenom market. Some products have efficacy and safety issues, are expensive to produce, and many exist in a loosely regulated environment. 💊 We also found a diverse pipeline of 196 candidates, but progress is slow moving. Further investment and more diverse actors are needed to push candidates through clinical development and scale up 🕒 Antivenoms save lives and will continue to play a key role in the treatment of SBE, but our data validates a shift towards a dual R&D agenda: focusing both on improving existing antivenoms in the short to medium-term, and on developing more cost-effective and safer, next-generation SBE therapeutics in the longer term. 🤝 Achieving the short- and long-term priorities will require more coordination and consensus. Established governance structures could help funders and researchers to align on an R&D agenda, prioritise a portfolio of work, and better understand the community’s capacities and needs. 📣 We’re seeing unprecedented levels of R&D funding and exciting scientific developments but we will soon be faced with a stark funding landscape. Advocacy and evidence will play a key role in engaging funders to ensure SBE stays on the agenda, and innovations can reach those who need them most. Read the article here: https://lnkd.in/gB2keeUR Thanks to the co-authors, José María Gutiérrez, Cecilie Knudsen, Abdulrazaq Habib, Maya Goldstein, Andrew Tuttle; and the Advisory Committee, Ian Cameron, Nicholas Casewell, Andreas Laustsen-Kiel, Matthew Lewin, Devin Sok and Julien Potet Funding: Wellcome Trust

Chronic #HepatitisDelta is a devastating viral disease with no approved treatments in the United States. I'm energized to see today's preliminary results from Vir Biotechnology, Inc.'s Phase 2 SOLSTICE clinical trial, evaluating mono- and combination therapies in the treatment of #HepatitisDelta. It's days like today that make me even more excited to come to work tomorrow. #PatientsAreWaiting https://lnkd.in/gHHhgdVS

Today is Rare Disease Day - the official international awareness raising campaign for the 300 million people currently living with one of the 6,000+ rare diseases worldwide. The primary goal of the campaign is to increase awareness amongst the general public and decision-makers about rare diseases and their influence on the lives of families living with these conditions. In the article below, BAP Pharma's Group Director of Medicines Access and UK General Manager - Rebecca Bibby - highlights our Medicines Access team's commitment to bridge the gap between patients and companies developing advanced medical treatments, providing access to combat rare diseases across the globe. https://lnkd.in/ej5q4BwU For more information on how BAP Pharma can support your medicines access needs, please contact us at bd@bappharma.com or visit our website at www.bappharma.com #rarediseaseday #raredisease #medicinesaccess #marketaccess #clinicaltrials #cellandgenetherapy #celltherapy #generaltherapy #biotech #medicine #pharmaceuticals #oncology #patientexperience #pharmacy #pharmacists #orphandrugs #biotechnology #nhs

ICYMI: Last Friday, the FDA announced the launch of a new #PilotProgram, called the Support for Clinical Trials Advancing Rare disease Therapeutics (#START), to help further accelerate the development of novel drug and biological products for rare diseases. Through this program, selected participants will have access to frequent advice and regular ad-hoc communication with FDA staff to address product-specific development issues, including #ClinicalStudyDesign, choice of control group, and fine-tuning the choice of #PatientPopulation. 🚀 This exciting new development is coming on the heels of the EveryLife Foundation for Rare Diseases publication on the costs of #DelayedDiagnosis of #RareDiseases (link in comments below). The START pilot program offers companies, especially those with innovative research into rare disease treatment options, greater access to the FDA at every step of the process. Considering the level of investment required through the #DrugDevelopment process and the amount of time lost with missteps, I am hopeful that it will accelerate the pathway to new and innovative treatments for patients with rare diseases. Here are some additional details about the START Pilot Program: ✅ Eligibility: The program is open to sponsors of novel drug and biological products for rare diseases (a small, limited number of participants yearly). The product must have a rare disease designation from the FDA and must be in the early stages of clinical development. ✅ Selected participants will be able to obtain frequent advice and regular ad-hoc communication with FDA staff to address product-specific development issues. 👇🏽 See the comment below for links to the FDA announcement and my post with the EveryLife report!

In the complex landscape of rare diseases, the key to commercial success often lies in effective collaboration and meaningful partnership with patients and PAGs. Experience shows that the most forward-thinking companies excel by evolving their approach from simply delivering products to actively collaborating with those patient communities they aim to help. The ZS Rare Disease Practice explores strategies for pharmaceutical companies in this challenging field in our recent publication, sharing the transformative power of leveraging patient insights to overcome commercial hurdles. Join us in recognizing the value of these partnerships, which bring not only a deeper understanding of the market but also much-needed relief and support to individuals facing rare diseases.  #raredisease #zs Rare disease care: Patient advocacy and transformative breakthroughs | ZS https://lnkd.in/eX32GfTn