Allelica, Inc’s Post

🎯 Challenges in rare disease drug development include small patient populations, limited disease knowledge and financial constraints. 💡 Innovative designs in clinical trial, potential multinational research collaborations and personalized treatments offer promising opportunities. 👀 In this article, #Alcimed explores these challenges and opportunities, emphasizing the need for continuous research and interdisciplinary collaboration to enhance the lives of those affected by rare diseases. #RareDiseases #Healthcare #ResearchAndDevelopment #Pharma #Innovation   https://lnkd.in/dKuhYKVt

Today is #RareDiseaseDay, and Norstella joins the global community in raising awareness for those affected by rare diseases. Our mission echoes this sentiment: to help bring life-saving therapies to market quicker—and help patients in need. Rare disease is one therapeutic area in which large pharma companies have seen significant investment in recent years, though many challenges remain. Norstella experts share their insights on the major trends and challenges currently impacting the rare disease landscape on our blog. Click to read it here: https://ow.ly/4eXt50QJ7wJ #Pipeline2Patient #RareDiseases #RareDiseaseAwareness #Pharma

Today, on #RareDiseaseDay, Citeline acknowledges this significant day dedicated to raising awareness and driving change for the 300 million people worldwide who live with rare diseases and their families and caregivers. Rare disease is one therapeutic area that has seen significant investment in recent years by large pharma companies, though many challenges remain. In this article Citeline and Norstella experts share their insights on how the life sciences industry is shaping up for a new era of patient recruitment and retention: https://ow.ly/Ji3550QJfSu. #Pipeline2Patient #RareDiseases #RareDiseaseAwareness #Pharma

Follow the link below to read some great insights from Toby Wilson Waterworth on deals associated with #rarediseases

Legend Biotech #ASH2023 #CARVYKTI® #CARTITUDE-4 #Abstract4866 PROs from the CARTITUDE-4 Study Showed Clinically Meaningful Improvements in Health-Related Quality of Life and Reductions in Multiple Myeloma Symptoms Following Treatment with CARVYKTI® (ciltacabtagene autoleucel) ---The as-treated population in CARTITUDE-4 demonstrated strong rates of progression-free survival and overall response ---Longer-term data from CARTITUDE-2 showed deep and durable responses in earlier lines of treatment among patients in Cohort A and Cohort B

The article below is free to view and includes comments/analysis from myself along with several of my colleagues about trends and challenges in the rare disease ecosystem. #rarediseaseday #rarediseases #orphandrugs

Spending the rarest of days (Leap Day) at the Rare Disease Clinical Trial Conference in Dublin, IE. Incredible group of investigators, patient advocates, clinicians and pharma industry representatives working to accelerate the development of effective treatments for (the 300 million!!) worldwide patients affected by rare diseases. A lot has been accomplished, especially since the passage of the U.S. Orphan Drug Act in 1983. There are currently more than 6,340 orphan drugs approved to treat 1,079 rare diseases. And with 80% of rare diseases having a genetic component, genetic testing and diagnostics are critically important But, we must do so much more. There are +/- 8,000 rare diseases, and nearly 7,000 of them have NO TREATMENT at all. And simple diagnostic capabilities are not available in low and middle income countries. So, if you haven’t recently, take today to recognize the patients and families that you know who are directly impacted by a rare disease. And please keep your eyes open for an opportunity to help. #RareDiseaseDay #RareDisease #RareDiseaseAwareness #RareTrials24 #PQMD #GlobalGenes #TheMaxFoundation #PartnerTherapeutics #TravereTherapeutics #TannerPharmaGroup #ImprovingLives

$1-Billion-dollar deals: A new era for rare disease treatments? In a landscape where only 5% of rare diseases have approved treatments, recent acquisitions signal a tidal shift. With companies like Ipsen, Chiesi Farmaceutici, and Amgen investing billions, the economics of rare disease treatments are evolving. Read my latest opinion piece in the link and comment here what your thoughts are on the current landscape for rare disease treatments. https://lnkd.in/ea5Da82b #BiotechInnovation #HealthcareInnovation #RareDisease #AmbroseHealthcare

ImmuPharma PLC (AIM:IMM) has made progress in the development of treatments for autoimmune diseases, particularly Systemic Lupus Erythematosus (SLE) and Chronic Inflammatory Demyelinating Polyneuropathy (CIDP), the company said in an update accompanying its interims. Unlike traditional treatments, the drug targets the malfunctioning immune system without suppressing it, which could broaden its use case beyond the two indications for which it is seeking approval. In SLE, a new phase III clinical trial is being prepared in collaboration with Simbec-Orion. This study will use a higher monthly dose of P140 compared to the previous evaluation completed in 2018, aiming to improve patient outcomes. More at #Proactive #ProactiveInvestors http://ow.ly/PUNI105I6iA

🧬 3 challenges and opportunities in rare disease drug development 🇪🇺 In Europe alone, approximately 36 million people are suffering from rare diseases, which is approximately equivalent to half the population of France. 🚨 There is no cure for most rare diseases, and the field suffers from gaps in medical and scientific understanding. Nevertheless, impressive progress has already been made in recent years and appropriate treatments and medical care have proven to enhance the quality of life and extend the lifespan of those affected. Our team explored three challenges and three opportunities for the current R&D of rare diseases: 🔴 Challenge 1: small patient populations exacerbate clinical trials 🔴 Challenge 2: limited knowledge of rare diseases impacts research strategies  🔴 Challenge 3: development of therapies for rare diseases isn’t financially feasible Despite these challenges, there are promising opportunities that could pave the way for the development of new therapies for rare diseases: 🟢 Opportunity 1: innovative technologies and designs in clinical trials 🟢 Opportunity 2: potential of multinational research collaborations  🟢 Opportunity 3: chances of personalized treatment approaches 💡 Want to learn more about the challenges and opportunities that could transform the landscape of rare disease research? Read our latest article here: https://lnkd.in/eNDuMZWU #healthcare #pharmaceuticals #pharmaceuticalindustry #raredisease #drugdevelopment