Life Science at RISE’s Post

G protein-coupled receptors (GPCRs) are the largest and most diverse group of membrane receptors in the body. 📡🧬 Many drugs and biologics target GPCRS, including allergy medicines such as antihistamines, pain relief drugs, and diabetes treatments. 💊 Still, around 75% of GPCR therapeutic targets are yet to be drugged, representing untapped opportunities for drug discovery and development to address numerous diseases. 🔍 We take a look at eight biotechs that have acquired funding in recent years to fortify their GPCR-focused pipelines, most of which have hit the clinic. 🏥 Read our latest article to find out! 👇 https://lnkd.in/dFSsArxA #GPCR #drugdiscovery #biotech #pharmainnovation #healthcareinnovation #biotechnews Confo Therapeutics | Domain Therapeutics | Escient Pharmaceuticals | Orion Biotechnology | Septerna | Structure Therapeutics | Tectonic Therapeutic, Inc. | Teon Therapeutics Inc.

Where does ELISA fall short? The ELISA (enzyme-linked immunosorbent assay) is the most common method for measuring and controlling host cell proteins (HCPs) in biologic drugs. However, ELISA has significant limitations that have caused project delays and even project terminations. In the upcoming US Pharmacopeia General Chapter <1132.1> Residual Host Cell Protein Measurement in Biopharmaceuticals by Mass Spectrometry, a USP expert panel has summarized the shortcomings of ELISA: [1] 👉 Polyclonal antibodies used in the immunoassay often have no or limited coverage to particular HCPs that are non-immunogenic or weakly immunogenic in the animals used to raise antibodies. 👉 The immunoassay may underestimate or overestimate levels of HCPs when individual HCP levels are very high and the antibody is limiting their detection or when an HCP is highly immunogenic in the immunized species dominating the ELISA signal. 👉 An ELISA assay generates a single value for total HCPs and does not distinguish the contribution of individual HCPs. 👉 The lack of specific identification prevents a knowledge-based risk assessment of particular HCPs. In addition, ELISA results depend on proprietary reagents that are tied to a product or production system, precluding industry-wide standardization of the method. In short, the limitations of ELISA—reliance on polyclonal antibodies with variable coverage, its inability to differentiate individual HCPs, and the lack of standardized reagents—can lead to inaccurate HCP levels and hinder data-driven risk assessments, potentially causing costly project delays. To overcome these challenges, the USP General Chapter <1132.1> will outline best practices for mass spectrometry (MS)-based HCP analysis, offering an orthogonal analysis for more precise identification and quantification of individual HCPs. To learn more about the new chapter on HCP measurement by MS, watch our latest webinar with the US Pharmacopeia: https://lnkd.in/dxZRjemm References: [1] USP Proposed Chapter <1132.1> Residual Host Cell Protein Measurement in Biopharmaceuticals by Mass Spectrometry https://lnkd.in/dxTuTsJ

Glycosylation for Therapeutic Applications Glycosylation has become a focus for therapeutic applications, particularly in the development of biologics, where glycan structure significantly affects the efficacy, stability, and safety of therapeutic proteins. One of the most prominent examples is the production of monoclonal antibodies (mAbs), where glycosylation affects their functions, including antibody-dependent cellular cytotoxicity (ADCC) and complement-dependent cytotoxicity (CDC). By optimizing glycosylation patterns, scientists can enhance the therapeutic activity of mAbs, making them more effective in treating cancer, autoimmune diseases, and infectious diseases. Glycoengineering is also making progress in vaccines. The development of glycosylated antigens could improve immune responses, leading to more effective vaccines. For example, glycan-based vaccines against bacterial pathogens target specific glycan structures on the bacterial surface, triggering a strong and targeted immune response. These glycan-modified vaccines have shown promise in preclinical and clinical trials. In enzyme replacement therapy (ERT) for lysosomal storage diseases, glycosylation is critical for delivering therapeutic enzymes to the correct cellular compartments. Modifying the glycosylation of these enzymes can enhance their uptake by cells, thereby improving therapeutic efficacy. Additionally, the potential of glycosylation in regenerative medicine is being explored, particularly in the development of glycosylated biomaterials that can promote tissue repair and regeneration. These innovations highlight the critical role of glycosylation in advancing therapeutic approaches and improving patient outcomes. Reference [1] Xueting Ren et al., International Journal of Biological Sciences 2024 (10.7150/ijbs.93806) [2] Peter Seeberger et al., Essentials of Glycobiology [Internet]. 4th edition 2022 (https://lnkd.in/emtsU2X3) #Glycosylation #Biologics #MonoclonalAntibodies #GlycoEngineering #Vaccines #EnzymeReplacementTherapy #RegenerativeMedicine #Therapeutics #Biopharmaceuticals #BiomedicalResearch

🧬 Biopharma Bite Size News - May 20th - 24th New $1.5B ADC manufacturing facility & 9 more updates...👇 Get these weekly → https://lnkd.in/gdjqWqiV 🤝 → 𝐌&𝐀 𝐚𝐜𝐭𝐢𝐯𝐢𝐭𝐲 HI-Bio & Biogen 🤝 Biogen to acquire HI-Bio for $1.8B acquisition will strengthen Biogen’s immunology portfolio. Acquisition is expected to close in Q3 2024. AstraZeneca  🤝 Invested $1.5B to build its first end-to-end ADC manufacturing facility in Singapore. This state-of-the-art facility will cover the entire ADC production process at a commercial scale and is expected to be operational by 2029.  — 🎯 → 𝐀𝐩𝐩𝐫𝐨𝐯𝐚𝐥𝐬 Amphastar Pharmaceuticals, Inc. 🎯 FDA approves albuterol sulfate for bronchospasm treatment and prevention. — 🔬 → 𝐍𝐞𝐰 𝐝𝐚𝐭𝐚 Arrowhead Pharmaceuticals  🔬 Ph1/2 trial of ARO-RAGE treatment in patients with asthma demonstrated a high level of gene knockdown, with serum reduction reaching up to 88%. Dyne Therapeutics  🔬 New data reported from ACHIEVE Ph1/2 trial of DYNE-101 in DM1 and DELIVER Ph1/2 trial of DYNE-251 in DMD demonstrating a significant impact on key disease biomarkers and improvement in multiple functional endpoints. Esperion 🔬 Bempedoic acid met the primary endpoint in a Ph3 trial in Japan for the treatment of hypercholesterolemia, demonstrating a statistically significant reduction in LDL cholesterol compared to placebo. Gilead Sciences  🔬 Interim Ph3 results reported from the seladelpar treatment in patients with PBC showing improved markers of liver disease progression and reduced itching. Citius Pharmaceuticals, Inc.  🔬 Mino-Lok antibiotic lock solution successfully met primary and secondary endpoints in a Ph3 trial, significantly reducing catheter-related bloodstream infections and microbial colonization in patients with central venous catheters. Zealand Pharma 🔬 Reported positive topline results from the DREAM Ph2 trial of its GLP-1/GLP-2 receptor dual agonist, dapiglutide. Low doses of dapiglutide led to a mean weight loss of up to 4.3% after 12 weeks and were well-tolerated, supporting its potential as a treatment for obesity. Cogent Biosciences 🔬 Reported positive updated data from the ongoing Ph3 PEAK trial at ASCO, showing a median progression-free survival of 10.2 months for bezuclastinib + sunitinib in patients with gastrointestinal stromal tumors (GIST). The combination therapy continues to show a promising safety profile. --- 👋 I'm Dominic Vacchiano, Co-founder of BiopharmIQ by Amp. We maintain biopharma data to help with your sales/marketing activities. 📰 Each week we share clinical/regulatory/funding news in an easy-to-digest format. #biotech #biopharma

‼️Ph. Eur. just released ‼️ New monographs on an anti-interleukin monoclonal antibody released for public consultation‼️ The #EuropeanPharmacopoeia (Ph. Eur.) has published two new draft monographs, Ustekinumab concentrated solution (3165) and Ustekinumab injection (3188), for public comment in this quarter’s issue of #Pharmeuropa (36.3). Following the adoption and publication of the monographs for #Etanercept (2895), #Infliximab concentrated solution (2928) and #Golimumab concentrated solution (3103), as well as the ongoing elaboration of a new monograph for #Adalimumab concentrated solution (3147), the #EuropeanPharmacopoeiaCommission (#EPC) has continued to build upon its work in the field of #therapeutic #monoclonalantibodies (#mAbs) – to date, predominantly focussing on TNF-alpha inhibitors -- and embarked on the development of standards for a mAb that targets another type of cytokines (i.e. #interleukins (#IL)), thereby addressing different complexities. 👀‼️Two new draft monographs for ustekinumab, a first-in-class therapeutic human #IgG1 kappa mAb that binds to IL-12 and IL-23, have been established using the #singlesourceapproach and reflect the considerations outlined in the EDQM’s scientific publication on Elaborating European Pharmacopoeia monographs for #biotherapeuticproteins using substances from a single source. https://lnkd.in/dkhW_-pA https://lnkd.in/drK3cnj3 A first wave of biosimilar product approvals for #ustekinumab has been recorded, with more biosimilars under development. ➡️ All interested parties are invited to review the drafts and submit comments on their technical content, via the appropriate channel. Stakeholder input will be key to refining and evolving the proposed monograph specifications, and to ensuring that they are and remain fit-for-purpose in a rapidly evolving multi-product market. The same issue of Pharmeuropa contains another new draft monograph, #Golimumab injection (3187), the first text on a medicinal product comprising the active substance described in Golimumab concentrated solution (3103), recently published in Ph. Eur. Supplement 11.6. Ustekinumab concentrated solution (3165) and Ustekinumab injection (3188) therefore form the second such “pair” of monographs for application in this dynamic field. ‼️➡️The commenting period runs until 30 September 2024. If you like this post follow me on LinkedIn

✅ CYT-108 is demonstrating exceptional tolerance in Cytonics Corporation's Phase 1 human clinical trial. All patients have received our 🧬 recombinant #protease inhibitor therapy for #osteoarthritis, no adverse events detected. We are excited to share this significant milestone in our Phase 1 clinical trial of CYT-108. All patients have received at least one dose of CYT-108, and 14 out of the 22 patients have received 2 doses (the maximum). Critically, the drug appears to be very well-tolerated - i.e., no drug-related adverse events have been reported so far. This safety data will provide critical support in our regulatory filings with the #FDA and #TGA. “This new safety data is very encouraging, as it clearly demonstrates that CYT-108 is well-tolerated after multiple doses. This information will play a critical role in convincing the regulatory bodies that CYT-108 is safe enough to proceed into larger, Phase 2 clinical trials. Truthfully, this result is not surprising, as CYT-108 shares 99% of the same DNA sequence with the naturally occurring A2M protein. We developed CYT-108 by genetically engineering a very small number of key #aminoacids in the region of the native #A2M protein that binds the cartilage-destroying protease enzymes. These small changes have a massive impact on CYT-108’s ability to neutralize proteases and prevent cartilage damage, with over 200% increase in potency compared to the naturally occurring A2M protein, while leaving the vast majority of the protein unchanged.” ~ Joey Bose, President & CEO 🌟 Upcoming Milestones: 🗸 We are on track to complete the final dosing in December 2024, after which all patients will be observed for an additional 3 months post-dose. 🗸 We anticipate the database lock in Q2 2025, after which no additional study data can be added to or modified. 🗸 The final Phase 1 clinical study report will be ready in Q3 2025, at which point we will be prepare the IND regulatory submission to the FDA. 🗸 We anticipate submitting our Investigational New Drug (IND) application to the FDA in Q4 2025, which is a prerequisite for launching Phase 2 clinical trials in the United States. ❤️ As always, a heartfelt Thank You to all of our shareholders who have enabled this remarkable success. We are defying the odds in this grassroots effort to beat #BigPharma and develop what may be the first and only disease-modifying therapy for osteoarthritis!

Immunotherapy Drugs Market Size To Hit USD 274.6 Billion by 2025 Download Free Sample Report-https://lnkd.in/dbhhQ9NQ The global immunotherapy drugs market in terms of revenue was estimated to be worth $163.0 billion in 2020 and is poised to reach $274.6 billion by 2025, growing at a CAGR of 11.0% from 2020 to 2025. Companies Working in the Market Mallinckrodt Pharmaceuticals    Allogene Therapeutics Aimmune Therapeutics Telesis Bio  Kyowa Kirin, Inc.- U.S.  Corbus Pharmaceuticals Takeda Oncology IGM Biosciences, Inc. MaxCyte, Inc. Octapharma Plasma, Inc. Celularity Inc. MAIA Biotechnology, Inc.   NKGen Biotech, Inc. Acumen Pharmaceuticals, Inc.    Octapharma ALK ABL Inc. Marker Therapeutics, Inc. Stallergenes Greer Adesis, Inc. Oxford Biomedica Myeloid Therapeutics Sengenics Pionyr Immunotherapeutics DIATER SimBioSys, Inc.    Minapharm Pharmaceuticals  Excelya BSP Pharmaceuticals S.p.A.   HAL Allergy Group  LETI Pharma Transgene LumaCyte PT LAPI LABORATORIES (OFFICIAL)  Angiocrine Bioscience, Inc. Octapharma Sweden Immunomic Therapeutics, Inc.    CytoImmune Therapeutics F-star, an invoX company Pheast Therapeutics

🚑 𝐇𝐨𝐬𝐩𝐢𝐭𝐚𝐥 𝐀𝐜𝐪𝐮𝐢𝐫𝐞𝐝 𝐏𝐧𝐞𝐮𝐦𝐨𝐧𝐢𝐚 (𝐇𝐀𝐏) 𝐃𝐫𝐮𝐠𝐬 𝐌𝐚𝐫𝐤𝐞𝐭: 𝐀 𝐆𝐫𝐨𝐰𝐢𝐧𝐠 𝐇𝐞𝐚𝐥𝐭𝐡𝐜𝐚𝐫𝐞 𝐏𝐫𝐢𝐨𝐫𝐢𝐭𝐲 🏥 𝐂𝐥𝐢𝐜𝐤 𝐇𝐞𝐫𝐞, 𝐓𝐨 𝐆𝐞𝐭 𝐅𝐫𝐞𝐞 𝐒𝐚𝐦𝐩𝐥𝐞 𝐑𝐞𝐩𝐨𝐫𝐭 https://lnkd.in/gV4ziZWF The Hospital Acquired Pneumonia (HAP) Drugs Market is a crucial segment of the pharmaceutical industry, driven by the rising incidence of pneumonia in hospital settings and the increasing threat of drug-resistant bacterial infections. Hospital-acquired pneumonia, also known as nosocomial pneumonia, occurs 48 hours or more after a patient has been admitted to a healthcare facility. The condition is associated with significant morbidity and mortality, particularly among patients in intensive care units (ICUs), and is one of the leading causes of infection-related deaths in hospitals. 𝐌𝐚𝐫𝐤𝐞𝐭 𝐓𝐫𝐞𝐧𝐝𝐬: Development of Novel Antibiotics: There is a strong focus on developing novel antibiotics that can overcome the resistance seen in common HAP-causing bacteria. These include drugs that target specific bacterial pathways and novel drug delivery systems for enhanced efficacy. Rising Use of Combination Therapies: Combination therapies are increasingly used to combat multidrug-resistant pathogens. These combinations may include two or more antibiotics, or a mix of antibiotics with adjunctive therapies, to ensure a broad-spectrum approach and prevent resistance. Focus on Rapid Diagnostics: The development of rapid diagnostic tools to quickly identify the pathogens responsible for HAP is gaining traction. Faster diagnostics lead to more targeted treatments, reducing the unnecessary use of broad-spectrum antibiotics and improving patient outcomes. Monoclonal Antibodies and Immunotherapy: Research is underway into monoclonal antibodies and other immunotherapies that can target bacterial toxins or enhance the body’s immune response to infections. These treatments could offer an alternative or adjunct to traditional antibiotic therapy. #Company Basilea Pharmaceutica Meiji Holdings Cubist Pharmaceutical Aridis Pharmaceutical Valneva Bayer GlaxoSmithKline Merck Group Achaogen AstraZeneca #Type Phase II Early Phase (Phase I & II) #Application Hospital Pharmacies Retail Pharmacies Drug Store E-Commerce #HealthcareInnovation #HAP #Pharma #HospitalCare #PneumoniaTreatment #InfectionControl #DrugDevelopment

CinFina Pharma Announces FDA Clearance of Investigational New Drug Application and First Participants Dosed in Phase 1 Trial of CIN-110 for the Treatment of Obesity CinFina Pharma, a CinRx Pharma portfolio company dedicated to advancing a portfolio of high-impact treatment options for obesity and metabolic diseases, today announced the U.S. Food and Drug Administration (FDA) has cleared the company’s Investigational New Drug Application (IND) for CIN-110, a PYY3-36 analog, allowing the first in-human clinical study to proceed. With the commencement of the trial, CinFina also announced the first cohort of participants has been dosed. The trial will evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics and immunogenicity of CIN-110 in a randomized, double-blind, placebo-controlled, single ascending dose study in otherwise healthy subjects with obesity. Dr. Jonathan Isaacsohn, MD, FACC, Founder and Chief Executive Officer at CinRx Pharma. “As demonstrated by promising preclinical efficacy data, CIN-110 has the potential to meet these needs and provide important therapeutic advantages to patients in monotherapy and combination settings, including durability of weight loss. We look forward to continuing to explore its safety and tolerability profiles in this important Phase 1 study.” “The preclinical program of CIN-110 underscores the potential for PYY to be a differentiated approach to treat obesity. This uniquely engineered molecule holds promise to expand the current therapeutic paradigm beyond GLP-1s with its selectivity, potency and half-life extension,” said Brian Murphy, MD, MPH, FIDSA, M.D., Chief Medical Officer at CinRx Pharma.

📢 Breaking News in the Fight Against Niemann-Pick Disease Type C (NPC) 📢 🌍 #NiemannPickDisease Type C (NPC) is a rare, progressive genetic disorder causing severe symptoms such as #hepatosplenomegaly, neurological disorders, and respiratory failure. In Germany and the UK, #miglustat (marketed as #Zavesca or #Yargesa) is the only approved treatment, while in the US, it is used off-label. 🔬 According to GlobalData's recent report, "Niemann-Pick Type C: Opportunity Assessment and Forecast – Update," the NPC market in the US, Germany, and the UK is expected to reach $220 million by 2031, driven by new pipeline therapies like arimoclomol citrate ER, acetylleucine, Trappsol Cyclo, and nizubaglustat. 🧬 Arimoclomol citrate ER, a heat-shock protein 70 amplifier, is poised to become the first approved drug for NPC in the US by the end of this year, with a Prescription Drug User Fee Act (#PDUFA) action date set for June 21, 2024. However, experts remain cautious about the efficacy of current pipeline therapies in stopping the progression of NPC. 💬 A key opinion leader (KOL) noted, "In the short term, within 52 weeks, there was a slowing of disease progression. However, starting at two years and beyond, the benefit becomes more muted. Patients and families are looking for a medication that stops, rather than just slows, disease progression." 🔗 #ZevraPharmaceuticals has shown the potential of combining arimoclomol with miglustat, enhancing drug efficacy. The future may see other pipeline drugs being used in combination with miglustat to improve outcomes for NPC patients. 💡 With 28 drugs in active development, the hope remains high for a breakthrough therapy that can revolutionize NPC treatment. The journey continues for an effective disease-modifying treatment to halt or reverse NPC progression. https://lnkd.in/eiK9GJAV https://lnkd.in/e9trhwA7 #pharmaceuticaltechnologu #RareDisease #NPC #NiemannPickDisease #Pharma #HealthcareInnovation #GlobalData #ZevraPharmaceuticals #Arimoclomol #Miglustat #DrugDevelopment #MedicalResearch #HopeForACure #globaldata 🖇️ Follow for more updates and insights into groundbreaking medical advancements!