Lycia Therapeutics’ Post

Tomorrow will be the first meeting of the FDA's newly formed, Genetic Metabolic Diseases Advisory Committee (GeMDAC). The GeMDAC will be discussing Zevra Therapeutics' NDA for arimoclomol for the treatment of Niemann-Pick Disease Type C (NPC). NPC is an ultra-rare neurodegenerative lysosomal storage disease which leads to neuronal death, uncontrolled seizures, and ultimately fatal dementia and early death by age 13 years for most patients. The unmet need for patients with NPC and their families is unimaginably urgent, with no FDA-approved treatments that can alter the course of the disease. In addition to a robust discussion of the specifics of the clinical data for arimoclomol to treat NPC, I am looking forward to observing how this new Committee works together to address the complex challenges of developing drugs for rare genetic metabolic diseases.

Longitude portfolio company Viridian Therapeutics, Inc. is increasing awareness about thyroid eye disease (TED) in women this #WomensEyeHealthMonth. “Women have a higher prevalence of many of the major vision conditions…with TED being five times more common in women than men.” Take a look at Viridian’s detailed TED overview, including details on common symptoms, understanding the TED journey, clinical studies, and more: https://lnkd.in/eZTGdQ9m

CEO Nadim Ahmed caught up with Brad Loncar of BiotechTV at #ASCO24 to discuss data Cullinan presented at this year’s conference & strategic expansion into autoimmune diseases. Check out the interview and learn more about Cullinan’s strategy and pipeline: https://bit.ly/455o5LY

The results from Arrowhead Pharmaceuticals' Phase 3 PALISADE study on plozasiran are truly impressive and could be a game-changer for patients with familial chylomicronemia syndrome (FCS). I'm excited by the potential impact this could have on a condition that currently has no approved treatments in the U.S. The involvement of experts like Gerald F. Watts and Bruce Given, M.D. adds significant weight to these findings. Their work not only showcases the power of RNAi therapeutics but also highlights how targeted innovation can meet critical, unmet medical needs. If plozasiran is approved, it could dramatically improve the quality of life for FCS patients, and it’s exciting to see this kind of progress in the field. #RNAi #FCS #research #Biotechnology

Galapagos CAR-T exits autoimmune space quoting "field is very crowded" (https://lnkd.in/dpbEasAz) while Kyverna Therapeutics's CAR-T for multiple sclerosis entered US trials for the first time. (https://lnkd.in/dYcFR_jS.) While very sensible to focus capital on more mature/promising programs, there is still plenty of space to grow in the CAR-T autoimmune space outside of Systemic lupus erythematosus (SLE). Would love to see more promising treatments for unmet medical needs on the market! #autoimmune #cartcelltherapy #cellandgenetherapy #clinicaltrials

#Lupus #SLE and #Sjogrens patients suffer an average of 4-6 years before receiving a diagnosis, seeing an average of 3-4 doctors on their journey. Sadly, many have permanent organ damage at the time of diagnosis. Shockingly, despite normal labs during disease activity and flares (only around 20% develop low complement levels), archaic labs are a huge part of the problem. It's easier to develop an expensive monoclonal antibody for lupus and get it FDA-approved than to approve new labs with the FDA or CMS. Learn more about these problems on April 27 at 10 AM PST (1PM EST) on the Facebook Lupus LA page. Click below to participate. Change cannot come unless the public and those in power know a problem exists. Let's fix the lab approval process. https://lnkd.in/e9z5EdZr Lupus LA

🎈 🎈 Check out this #HighlyCitedPaper# by Olguța Anca Orzan et al: "An Insight on the Possible Association between Inflammatory Bowel Disease and Biologic Therapy with IL-17 Inhibitors in Psoriasis Patients" You can find it at: https://lnkd.in/gvyBPCcV

Featured Presentation: RIPK1 Inhibitors and Inflammation with Domagoj Vucic from Genentech. I will discuss the role of RIP1 in inflammatory bowel disease as well as its role in tissue damage for other related diseases. Progress on RIP1K inhibitors will also be included. https://lnkd.in/eCUAustN Save $150 with Discount code SPK150. #drugdiscoverychemistry

From viral and bacterial #pneumonia and #sepsis, to acute kidney injury, hemorrhagic shock, #stroke, and complications associated with #diabetes, vascular dysfunction is a foundational mechanism for an array of the most devastating and pervasive disease states affecting patients worldwide. Recognizing the potential far-reaching impact and scale of opportunity thereby presented by vascular normalization strategies, TIAP and Sunnybrook came together in 2014 and created Vasomune Therapeutics, Inc. With its made-in-Canada solution currently in the midst of the ongoing fight against infection-induced respiratory distress (via a Phase IIa trial), and with a remarkable opportunity to subsequently expand its impact to additional areas of immense unmet global need, Vasomune is clearly an Ontario life sciences company to keep your eye on! Read more about the Vasomune Therapeutics, Inc. story at: https://t.ly/vMuWE TIAP would like to acknowledge the support of the Federal Economic Development Agency for Southern Ontario | Agence fédérale de ... in enabling this success story. Brian Jahns

#GeographicAtrophy (GA) is an advanced, vision-threatening form of age-related macular degeneration (AMD) affecting approximately five million individuals worldwide. While there are currently no approved therapeutics for treatment, our #JNJRetina team continues to work to develop innovative solutions for those living with GA. Learn more about our commitment to helping patients with blinding retinal diseases restore and preserve their vision: https://bit.ly/4aO7ZaJ