bioSeedin’s Post

We’re cheering on the recently announced participants in the FDA's START Pilot Program! Aiming to accelerate novel drug and biologic development in the rare disease space, the FDA reviewed applications earlier this year and accepted the following companies into the Program: Larimar Therapeutics Inc. (Friedreich’s Ataxia) Grace Science, LLC (NGLY1 Deficiency) Myrtelle (Canavan Disease) Calico Life Sciences (Vanishing White Matter Disease) Denali Therapeutics (Sanfilippo Syndrome Type A) Neurogene Inc. (Rett Syndrome) The START Pilot Program was designed to enhance communication with FDA staff around clinical study design, control groups, patient population, nonclinical information, and product characterization. This is one of several programs announced by the FDA in recent months including: Rare Disease Endpoint Advancement (RDEA), CMC Development and Readiness, and the Platform Technology Designations Program. At DKP we are hopeful that these initiatives will improve support for development and eventual approval of groundbreaking treatments, providing much-needed hope to patients. #FDA #raredisease #rarediseases #genetherapy #neurodegenerative #pediatricraredisease #rettsyndrome #sanfilipposyndrome #vanishingwhitematterdisease #canavandisease #ngly1deficiency #friedreichsataxia

Ventus Therapeutics announced that its partner, Novo Nordisk, has successfully dosed the first participant in a Phase 1 clinical study for NNC6022-0001 – an oral NLRP3 inhibitor licensed by Novo Nordisk in September 2022. NLRP3 inhibitors have potential in a wide variety of cardiometabolic diseases, including obesity, metabolic dysfunction-associated steatohepatitis (MASH), and chronic kidney disease. Read: https://lnkd.in/egk6PPbV #sciencesdelavie #biotechnology #biotechnology #innovation #innovations #research #sciences #medicaments #research #drugresearch #laboratories #labs #chemistry #chemistry #biology #biologie #biopharma #rna #rnatherapeutics #arn #revolution #happy

🌊 This will be an interesting one to watch in the anti-obesity pipeline, from Wave Therapeutics, potentially challenging the #GLP1 class: "Presentation at the Obesity & Weight Loss Drug Development Summit to review Real World Evidence for the GLP-1 class and the opportunity for Wave’s INHBE program as a best-in-class approach for obesity designed to induce fat burning and preserve muscle mass with once or twice-annual subcutaneous administration: Citing very #competitive and #differentiated benefits of: 📅 Less frequent, likely to be more favorable, dosing only once or twice a year, via SC ROA 💪 Fat-burning mechanisms without sacrificing muscle mass. "Presentation at the Obesity & Weight Loss Drug Development Summit to review Real World Evidence for the GLP-1 class and the opportunity for Wave’s INHBE program as a best-in-class approach for obesity designed to induce fat burning and preserve muscle mass with once or twice-annual subcutaneous administration." #WaveTherapeutics #antiobesity #Biotech #Biopharma #RWE #Healthcare

Ventus Therapeutics announced that its partner, Novo Nordisk, has successfully dosed the first participant in a Phase 1 clinical study for NNC6022-0001 – an oral NLRP3 inhibitor licensed by Novo Nordisk in September 2022. NLRP3 inhibitors have potential in a wide variety of cardiometabolic diseases, including obesity, metabolic dysfunction-associated steatohepatitis (MASH), and chronic kidney disease. Read:  https://lnkd.in/ek4AtcjJ #sciencesdelavie #biotechnology #biotechnology #innovation #innovations #research #sciences #medicaments #research #drugresearch #laboratories #labs #chemistry #chemistry #biology #biologie #biopharma #rna #rnatherapeutics #arn #revolution #happy

Free searchable database of therapeutic biomarkers, TheMarker. Includes 103 surrogate endpoints, 104 monitoring/response, 218 pharmacodynamic, 624 safety. And 15893 response-predictive. Includes tissue/organ-specific profiles. Developed in China at Zhejiang University by Yintao Zheng et al. https://lnkd.in/eSDEbXq9 https://lnkd.in/ez3Haq3s

We're excited to introduce our latest service, the Cardiomyocyte Contraction Assay. Here's what our service offers: ✅ Drug Screening: Evaluate pharmacological compounds' effects on cardiomyocyte function, facilitating rapid screening of potential therapeutics for cardiovascular diseases. ✅ Mechanistic Insights: Uncover the underlying molecular mechanisms of drug candidates by studying their impact on cardiomyocyte contractility, providing critical insights into their mode of action. ✅ Safety Assessment: Assess the cardiotoxic effects of drugs and environmental toxins early in the drug development process, ensuring the safety and efficacy of your compounds. ✅ Flexibility: Our Cardiomyocyte Contraction Assay is available for both Rat Primary Cardiomyocytes and hiPSC-CMs, providing flexibility and adaptability to suit your research needs. #cardiomyocytes #safetypharmacology #drugdiscovery #contraction

The global acquired aplastic anemia market is expected to reach USD 10,246.1 Million by 2030 at 4.20% CAGR during the forecast period 2022-2030, according to Market Research Future Get More Details at: https://lnkd.in/dE9Qbhy2 #Acquired #Aplastic #Anemia is a rare hematologic disorder characterized by the failure of the bone marrow to produce an adequate number of blood cells. Unlike the inherited form, acquired aplastic anemia develops later in life and is often triggered by factors such as exposure to toxins, certain medications like #chemotherapy drugs, radiation therapy, or viral infections such as hepatitis or Epstein-Barr virus. The condition leads to a deficiency in red blood cells, white blood cells, and platelets, causing symptoms such as fatigue, #weakness, increased susceptibility to infections, easy bruising, and prolonged bleeding. #Diagnosis typically involves blood tests to assess blood cell counts and a bone marrow biopsy to confirm the diagnosis and determine the severity. Treatment options vary depending on the severity of the condition and may include blood transfusions, medications to stimulate bone marrow function, #immunosuppressive therapy, or, in severe cases, bone marrow transplantation. Close monitoring by healthcare professionals is essential to manage symptoms effectively and improve quality of life. The acquired aplastic anemia market includes some of the key players such as Pfizer Inc. (U.S.), Bayer AG (Germany), Novo Nordisk AS (Dernmark), Shire (Republic of Ireland), SOBI (Sweden), Octapharma (Switzerland), CSL Limited (Australia), Amgen Inc., GSK plc., bluebird bio #BoneMarrowFailure #BloodDisorder #RareDisease #Healthcare #BloodTransfusion #MarketResearchFuture #marketresearch #consultingservices #consultingfirms #consultingbusiness #mrfr

Roche's venture into the obesity sector is showing promising outcomes, evidenced by the initial clinical results post its acquisition of Carmot Therapeutics for $2.7 billion. Carmot, a clinical-stage biotech firm dedicated to transformative therapeutics for metabolic diseases like obesity and diabetes, caught Roche's attention with its portfolio of potential game-changers, poised to compete with industry leaders like Eli Lilly and Novo Nordisk. Notably, Roche acquired CT-388, an injectable dual GLP-1/GIP receptor agonist already in phase 1b trials. Recently released 24-week data from CT-388's trial demonstrated an average placebo-adjusted weight loss of 18.8%, with remarkable results across participants. These findings suggest CT-388's competitive edge in a crowded field. Investors responded positively, driving a 4% increase in Roche’s shares. Roche's morning release highlighted CT-388's tolerability and its potential for both obesity and type 2 diabetes. Further analysis showed promising results, especially among pre-diabetic participants. Roche's acquisition not only strengthens its presence in the obesity sector but also provides access to Carmot’s diverse portfolio of incretins, enhancing Roche’s R&D efforts across cardiovascular and metabolic diseases. This acquisition comes at a time when obesity poses a significant global health challenge, with associated comorbidities like type 2 diabetes and cardiovascular diseases. Scientific advances in incretin research offer promising avenues for treating obesity and related conditions, marking a shift in therapeutic approaches. #api #science #drugdiscovery #pharma #obesity #peptide #ozempic #diabetes #clicnicaltrials

🌟 Advancing Drug Discovery in Liver Disease: The MASH CALL Initiative 🌟   🔍 Identifying the best candidates early on to minimize attrition rates is one of the biggest challenges in #drugdiscovery. To address this, there's an increasing need for advanced in vitro 3D models that can reliably predict clinical outcomes. These models need to be #scalable and #costeffective, compatible with #automation, and capable of delivering diverse readouts for complex phenotypic analysis. Our 3D InSight™ MASH model consists of primary human ✔️hepatocytes, ✔️Kupffer cells, ✔️endothelial cells, and ✔️stellate cells. These healthy liver spheroids are exposed to steatotic and pro-inflammatory media conditions, which over 🗓10 days result in the main hallmarks of human MASH (steatosis, inflammation, and fibrosis). At InSphero we take the next step in drug discovery for MASH by launching the MASH CALL Initiative – an exciting opportunity to push the boundaries of what's possible in drug discovery and bring new, effective treatments to the market faster 🚀   Join the MASH CALL by 🗓 September 6 and let's work together to revolutionize the future of MASH therapeutics! 💊 Learn more via the link in comments 👇 Francisco Verdeguer Radina Kostadinova, PhD Jesus Francisco Glaus Garzon Angelina Freitag Joel Zvick Philip Vonschallen Paul Edgard Clémençon Arumugham (Ragoo) Raghunathan Bärbel Ulmer Christine Schwenk Sue Grepper, PhD Madhu Lal Nag, MBS PhD Jan Lichtenberg Rositsa Hadzhipetrova Tina Žel #DrugDiscovery #Innovation #Healthcare #MASHCALL #Pharma #3DModels #PhenotypicAnalysis

𝐀𝐬𝐭𝐫𝐚𝐙𝐞𝐧𝐞𝐜𝐚 𝐭𝐨 𝐚𝐜𝐪𝐮𝐢𝐫𝐞 𝐀𝐦𝐨𝐥𝐲𝐭 𝐏𝐡𝐚𝐫𝐦𝐚 𝐟𝐨𝐫 $1.05 𝐛𝐢𝐥𝐥𝐢𝐨𝐧 𝐭𝐨 𝐬𝐭𝐫𝐞𝐧𝐠𝐭𝐡𝐞𝐧 𝐢𝐭𝐬 𝐩𝐢𝐩𝐞𝐥𝐢𝐧𝐞 𝐟𝐨𝐫 𝐫𝐚𝐫𝐞 𝐝𝐢𝐬𝐞𝐚𝐬𝐞𝐬, 𝐩𝐚𝐫𝐭𝐢𝐜𝐮𝐥𝐚𝐫𝐥𝐲 𝐡𝐲𝐩𝐨𝐩𝐚𝐫𝐚𝐭𝐡𝐲𝐫𝐨𝐢𝐝𝐢𝐬𝐦. 𝐒𝐭𝐫𝐚𝐭𝐞𝐠𝐢𝐜 𝐀𝐜𝐪𝐮𝐢𝐬𝐢𝐭𝐢𝐨𝐧 𝐑𝐚𝐭𝐢𝐨𝐧𝐚𝐥𝐞: - 𝐀𝐝𝐝𝐫𝐞𝐬𝐬𝐢𝐧𝐠 𝐏𝐢𝐩𝐞𝐥𝐢𝐧𝐞 𝐆𝐚𝐩𝐬: AstraZeneca, particularly through its Alexion rare disease division, aims to bolster its portfolio, especially in late-stage treatments for rare endocrine disorders, an area where Amolyt's offerings align well. - 𝐀𝐦𝐨𝐥𝐲𝐭'𝐬 𝐔𝐧𝐢𝐪𝐮𝐞 𝐎𝐟𝐟𝐞𝐫𝐢𝐧𝐠: Amolyt's key asset, eneboparatide, a Phase III drug for hypoparathyroidism, fills a crucial niche in the market, providing a novel approach that could improve patient outcomes and reduce reliance on current treatments. - 𝐒𝐭𝐫𝐚𝐭𝐞𝐠𝐢𝐜 𝐄𝐱𝐩𝐚𝐧𝐬𝐢𝐨𝐧: Beyond acquiring a promising drug, AstraZeneca gains specialized knowledge and a foothold in the rare endocrinology sector, paving the way for future growth and potential further acquisitions. Patients with hypoparathyroidism cannot produce enough parathyroid hormone, vital for controlling blood calcium and phosphorus levels. This leads to low levels of calcium and high levels of phosphorus in the blood, with symptoms including muscle cramps, pain and twitching in addition to complications such as chronic kidney disease. Injected subcutaneously every day, 𝐀𝐦𝐨𝐥𝐲𝐭’𝐬 𝐞𝐧𝐞𝐛𝐨𝐩𝐚𝐫𝐚𝐭𝐢𝐝𝐞 is designed to activate a cell surface protein called parathyroid hormone receptor 1 (PTHR1), mimicking the function of the hormone. Source: AstraZeneca PR #astrazeneca #amolyt #pharma #competitiveintelligence