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Are these siRNA companies on your radar? 🧭 Currently valued at around $12 billion, the market for siRNA therapeutics is expected to grow at a CAGR of 17.5% to reach $39 billion by 2030. This rapid expansion is driven by advancements in RNAi technology, its specificity in gene silencing, and the successful approval and commercialization of several siRNA-based drugs in recent years. 📈 Discover some of the companies that matter in the siRNA field! ⬇️ https://lnkd.in/dcyKJeKi #siRNA #RNAi #genetherapy #biotech #pharmaceuticals #healthcareinnovation #drugdevelopment #geneticmedicine #RNA #siRNAtherapeutics #biotechnews #medicalresearch #marketgrowth Alnylam Pharmaceuticals | Aro Biotherapeutics | Arrowhead Pharmaceuticals | Dicerna Pharmaceuticals, Inc. | Novo Nordisk | DTx Pharma, A Novartis Company | Novartis | Eleven Therapeutics | Switch Therapeutics | Wave Life Sciences

[update] Vertex Pharmaceuticals will use Orum Therapeutics’ targeted protein degradation (TPD²®) technology platform Orum Therapeutics has granted Vertex Pharmaceuticals rights to conduct research using its TPD² approach to developing DACs, next-generation versions of antibody-drug conjugates (#ADCs) which use antibodies to precisely deliver small molecule targeted protein degrader payloads to cancer cells and other targeted biological therapies. Vertex has agreed to pay Orum $15 million upfront, plus option payments and milestones potentially totaling up to $310 million per target for up to three targets—up to $930 million total. In September 2023, Orum sold its early clinical phase program to develop ORM-6151 to Bristol Myers Squibb (BMS) for $100 million upfront and an additional up to $80 million in milestone payments. For more info 👇🏻 #ADC #kbio #biopharmaceutical #innovative #drugdevelopment #koreabio

💊💉 𝑰𝒏𝒅𝒖𝒔𝒕𝒓𝒚 𝑵𝒆𝒘𝒔 💉💊 After a dip in the number of new therapies approved by the FDA in 2022, last year saw the second largest number of approvals in the last 30 years. 55 novel drugs were approved by the FDA in 2023, including 5 new gene therapies, hinting at a much anticipated bounce back for the industry. How will 2024 fair in comparison? #pharmaceuticals #biopharmaceuticals #FDA #industrynews #recruitment

ExoXpert, a CDMO launched by EXO Biologics to service increasing worldwide demand for exosomes - https://lnkd.in/gw_ad42J EXO Biologics  SA, a Belgian biotech company committed to developing biopharmaceuticals using exosomes to treat rare diseases with high unmet medical needs, today announces the worldwide launch of ExoXpertTM, a contract development and manufacturing organization (CDMO) specializing in exosomes. ExoXpert offers a MSC-based exosome manufacturing platform used in European clinical trials. ExoXpert is a wholly owned subsidiary of EXO BiologicsTM. “The potential of exosomes continues to be demonstrated across the cell and gene therapy sector. As a result, exosome-based clinical trials have the potential to significantly increase in number, if industrial supply of exosomes can be met. Currently there is a lack of exosome production, manufacturing specialists and facilities to supply exosomes for both R&D and clinical trials,” said Romain du Hecquet de Rauville, Chief Business Officer, EXO Biologics. Read more at: https://lnkd.in/gw_ad42J #exosomes #researchanddevelopment #stemcells

ExoXpert, a CDMO launched by EXO Biologics to service increasing worldwide demand for exosomes - https://lnkd.in/gbGt4WNy EXO Biologics  SA, a Belgian biotech company committed to developing biopharmaceuticals using exosomes to treat rare diseases with high unmet medical needs, today announces the worldwide launch of ExoXpertTM, a contract development and manufacturing organization (CDMO) specializing in exosomes. ExoXpert offers a MSC-based exosome manufacturing platform used in European clinical trials. ExoXpert is a wholly owned subsidiary of EXO BiologicsTM. “The potential of exosomes continues to be demonstrated across the cell and gene therapy sector. As a result, exosome-based clinical trials have the potential to significantly increase in number, if industrial supply of exosomes can be met. Currently there is a lack of exosome production, manufacturing specialists and facilities to supply exosomes for both R&D and clinical trials,” said Romain du Hecquet de Rauville, Chief Business Officer, EXO Biologics. Read more at: https://lnkd.in/gbGt4WNy #exosomes #researchanddevelopment #stemcells

Stories making headlines today in the biopharma industry: GSK is fast-tracking the development of its Blenrep treatment for multiple myeloma, ahead of its initial 2025 guidance. Plans are in place to submit to US, European, and Japanese regulatory bodies in the second half of 2024. GSK's confidence stems from promising results in the DREAMM-7 trial. Pfizer reported slight modifications to its pipeline in Q1. The firm discontinued the regulatory development of gene therapy VTX-801, a treatment for the rare inherited disorder Wilson’s disease, and two alternative applications for ulcerative colitis drug Velsipity. Nonetheless, Pfizer's pipeline saw the addition or advancement of 10 programs, with five receiving approvals in the US and EU. In another development, Astellas Pharma and Poseida Therapeutics have announced their second collaborative deal, focusing on solid tumor cell therapies. Subsidiary firm Xyphos Biosciences will use a Poseida CAR-T construct to develop two CAR-T therapy candidates. Editas Medicine and Bristol Myers Squibb extended their collaboration on gene-edited alpha-beta T cell therapies into 2026. In related news, KalVista Pharmaceuticals is seeking a partner for its program in hereditary angioedema, as it plans to discontinue work on its factor XIIa program. GSK's operating profit growth forecast for the year increased to 9-11%, following a successful Q1 2024 driven by its vaccine portfolio. The firm continues to focus on strengthening its innovative vaccine portfolio, including Shingrix and Arexvy. Tech firm Real Chemistry unveiled IRIS, a real-time data dashboard combining clinical trial tracking with consumer behavior and market access news. Initially for GLP-1 drug development entities, IRIS could also serve the fitness, food, and beverage sectors. Neurocrine Biosciences received approval for a 'Sprinkle' variant to its Ingrezza drug, designed for patients with difficulty swallowing. Meanwhile, Piction Health secured $6 million in funding to decrease wait times for skin conditions. Lastly, Eli Lilly halted trials for Verzenio, Jardiance, and a gene therapy from Prevail Therapeutics. #pharma #biotech #topstories #biodatastudio

RNAi-based drug design: considerations and future directions RNA interference (RNAi), a post-transcriptional gene regulation, is revolutionizing pharmaceutical development. Recently, the FDA approved several siRNA drugs targeting the liver, such as patisiran, givosiran, lumasiran, and inclisiran, for rare diseases. Synthetic siRNAs that trigger RNAi can potentially make any gene druggable by precisely silencing with huge potency and durability. However, significant challenges, particularly in delivery, have hampered bringing these innovative drugs to patients. While various siRNA drug classes are undergoing clinical trials, their effectiveness in treating diseases outside the liver (like neurological conditions) remains limited, necessitating ongoing advancements. This review explores key considerations and future directions for designing therapeutic siRNAs, focusing on aspects like chemical modifications, computational tools, delivery strategies, and meticulous target selection – all critical for achieving therapeutic success. https://lnkd.in/eqTERxwr #genetics #genomics #precisionmedicine #genomicmedicine #genetherapy #rna #rnatherapeutics #therapeutics #liver #hepatology #brain #neurology #sirna #drugdiscovery #drugdevelopment #drugdelivery #pharma #metabolism #raredisease #biotechnology #molecularbiology #innovation #research #science #sciencecommunication

Stories making headlines today in the biopharma industry: Roger Perlmutter, former research leader at Amgen and Merck, is now the CEO of Eikon Therapeutics. With Eikon's use of Nobel-winning microscope technology, they aim to observe the real-time mechanics of cells at the protein level to discover new drug targets. Perlmutter believes that current drug development lacks understanding of the intricate workings of the human body. Roche and Remix Therapeutics have entered into a deal worth up to $42 million, including a $60 million financing round. Remix Therapeutics focuses on modulating mRNA and boosting RNA production with their oral small molecules. Boehringer Ingelheim has partnered with Suzhou Ribo Life Science and Ribocure Pharmaceuticals to develop siRNA therapies for MASH. This potentially lucrative deal could be worth up to $2 billion. Agios Pharmaceuticals' oral drug candidate for thalassemia patients, mitapivat, has shown promising results in a late-stage study. Regulatory activities are set to begin by the end of the year. Goldman Sachs has closed a $650 million life sciences fund aimed at financing advancements in various healthcare fields. CG Oncology, a company specializing in drugs for bladder cancer, has filed for an IPO as the biotech market rebounds. Investment firm The Column Group has made a take-private bid for NGM Bio, a struggling biotechnology company with a Phase II candidate for treating NASH. Invivyd has requested emergency use authorization for a Covid-19 preventative antibody, particularly for immunocompromised individuals. Novartis is strengthening its neurologic gene therapy efforts through a partnership with Voyager, further committing to finding new treatments for neurologic conditions. #pharma #biotech #topstories #biodatastudio

#News: Abzena , a leading end-to-end integrated #CDMO for #complexbiologics and #bioconjugates, has announced the launch of its enhanced #bioassayplatform EpiScreen® 2.0, a comprehensive suite of assays that predict and evaluate potential risks of #preclinical #immunogenicity in #protein, #antibody, and #genetherapy therapeutics. The next generation tool provides a better immunogenic assessment that is highly sensitive, multi-parametric, and data-rich, which ultimately improves candidate selection and de-risks early phase development. Dr Campbell Bunce PhD MBA , CSO of #Abzena said, “Understanding a lead drug candidate’s immunogenicity risk profile and how we can mitigate it is a key step in advancing new drugs from discovery to clinical trials. We developed EpiScreen® 2.0 because we understand that there are many factors that contribute to the immunogenicity risk of a drug, especially with the next-generation therapeutics being developed like Antibody-drug conjugates (ADCs). Read more from Read Magazine 👇🏽 https://lnkd.in/edMsnYXp

FDA Issues Comprehensive Guidance for Industry and Academic Sponsors Developing Ex Vivo-Manufactured CAR T Cell Products in Oncology: Expert Recommendations for CMC, Pharmacology, and Clinical Trial Design The guide includes recommendations tailored to both autologous and allogeneic CAR T-cell products. In addition, it provides guidance on comparative analytical studies for CAR T-cell products. Although the focus is on CAR T-cell products, certain information and recommendations may also be relevant for other genetically modified lymphocyte products, such as CAR natural killer (NK) cells or T-cell receptor (TCR)-modified T-cells. It is important to note that for these related product types, further considerations beyond those suggested in this guidance may be required depending on the product and manufacturing process. https://lnkd.in/ePCkxmgh https://rea.works/