SpliceBio’s Post

The latest episode of our #OptimumPerspectivesPodcast is now live. #rarediseaseday2024

LYME AWARENESS MONTH DETROIT, Michigan, May 10th, 2024 -- Flightpath Biosciences Inc., a clinical-stage life sciences company developing therapeutics to treat infectious diseases, announced today that the first healthy volunteer participants have been dosed in its Phase I clinical trial of FP-100 (hygromycin A), an orally-available, narrow-spectrum antimicrobial drug candidate. The single center, prospective, randomized, double-blind, placebo-controlled study of single-ascending doses and multiple-ascending doses of FP-100 administered orally will include a maximum of 72 healthy adult volunteers to evaluate the safety, tolerability and pharmacokinetic profile of the drug. https://lnkd.in/gkPkFDep Invest Detroit Michigan Rise Ann Arbor SPARK Bay Area Lyme Foundation Global Lyme Alliance Project Lyme LymeDisease.org Illumina Ventures #LymeDisease #Lyme #tickbornedisease

SciRhom Secures EUR 63 Million Series A Financing Round to Accelerate iRhom2-targeting Therapies in Autoimmune Diseases. Financing used to accelerate lead therapeutic strategy toward clinical POC and to broaden the therapeutic value of the iRhom2 strategy https://lnkd.in/er3jsV4P #biotech SciRhom #autoimmune_diseases

Join our cohosts in unpacking the impact of Medicare Drug Price Negotiation on patient care in this 360it breakout conversation. They discuss the challenges in accessing innovative treatments for diseases like cancer and AiArthritis, emphasizing the urgency of finding the right treatment. Get involved with AiArthritis and others to advocate for a healthcare landscape that prioritizes patient needs! Listen to the full episode here: https://lnkd.in/eZipWyU5 ALLIANCE FOR SAFE BIOLOGIC MEDICINES #biosimilars #IRA #360it

Looking forward to the World Orphan Drug Congress this week, an important intersection between rare disease patients, drug developers, and regulators! 💊 We're glad to be a part of this discussion at Sano Genetics, bridging the gaps between patient recruitment, biomarker testing, and engagement to improve access to life saving therapies. Our Head of Partnerships and Patient Advocacy, Lindsey Wahlstrom-Edwards, will be joining a roundtable on the 25th focused on strategies to better support adults living with rare conditions, including tackling the barriers to genetic testing. If you're around, feel free to reach out to myself or Lindsey for a chat. #WorldOrphanUSA #clinicaltrials #precisionmedicine

Data sharing and Alzheimer’s Disease—learn more about it at the panel below:

How is biotech tackling myasthenia gravis? 🧬 Myasthenia gravis is a chronic autoimmune disorder where antibodies destroy the communication between nerves and muscle. This results in weakness of the skeletal muscles. 🎙️ Labiotech was in conversation with Dana Vigier, MD, head of Central Eastern and Northern Europe at Alexion Pharmaceuticals, Inc., to discuss how the disease is being treated, and what is in the pipeline for future treatment. 🏥 🎗️ As we observe Myasthenia Awareness Month in June, tune into the latest episode of the Beyond Biotech Podcast to find out more about future treatments for the disease! 👇 https://lnkd.in/e4T8szcg Myasthenia Gravis Foundation of America, Inc. | Myasthenia Gravis Association | Myasthenia Gravis Assoc | Myaware (Formerly Myasthenia Gravis Association) #Biotech #Raredisease #Neuromusculardisease #Research #Lifesciences #Myastheniagravis #Autoimmunedisorder #Clinicaltrials

Exciting news in the world of ocular therapies! Join OKYO Pharma's Key Opinion Leader event on April 9th discussing Phase 2 trial data for dry eye disease. Efficacy results will be unveiled on March 22nd. Don't miss out on this insightful session led by Dr. Jay Pepose and Dr. Anat Galor. Register now to learn more about OKYO Pharma's innovative approach to treating inflammatory dry eye disease. #OKYOPharma #DryEyeDisease

Today is Rare Disease Day at The National Institutes of Health! Stream the conference live here https://buff.ly/49MRWtI. The agenda is filled speakers from Congress, the FDA, Industry, Academia, and patient advocates all coming together to discussing ways to accelerate development of new therapeutics for this underserved population. We are proud to have several members of our team presenting at the event, specifically: James J. Hickman, Ph.D. will be speaking on the main stage at noon ET on how Microphysiological Systems (MPS) are currently supporting rare disease therapeutic development. Christopher Long, Kaveena Autar, and Xiufang Guo will be presenting posters on the following topics, respectively: "Implementation of a Human Cell-Based Malaria-on-a-Chip Phenotypic Disease Model for Drug Efficacy Evaluation" "Evaluation of Long-Term Potentiation and Drug Efficacy in a Familial Alzheimer’s Disease hiPSC-Cortical Neuron Microphysiological System" "A human-based functional NMJ system for personalized rare disease modeling and drug testing" #NIH #RareDiseaseDay #MPS #RDDNIH #OrganOnAChip #HumanOnAChip

Don't miss our keynote presentation by Dr. Daniel O'Connor. He will discuss new perspectives on how the regulatory system can be used to advocate for patients and individualized therapeutics in the rare disease ecosystem. Register today! https://lnkd.in/gzaxRX8q #N1CAnnualMeeting #IndividualizedMedicine #GlobalHealthPerspective #RareDiseases #PersonalizedMedicine #RegulatorySystem