PolTREG S.A.’s Post

B cells as drug factories Companies are engineering B cells to produce gene-encoded medicines, but clinical proof of concept is needed to convince the doubters that this platform can deliver. Early observations from the world’s first clinical trial of B cell ‘biofactories’, engineered to produce therapeutic proteins continuously inside a person’s body, has given the field grounds for optimism. These B cells are taken from a patient and engineered in the lab such that, once differentiated into antibody-producing plasma cells — how they would normally respond to invading pathogens — they instead make therapeutic proteins. These medicines made in situ require neither immunosuppression nor lymphodepletion and could offer developers a way to deliver genetic cargoes across a whole range of conditions that is safer and less cumbersome than current approaches. https://lnkd.in/e9iytCNj

A thought-provoking HBR article on the arrival of advanced therapeutic modalities (ATMs). If there is something I’ve learned working in CGT, it is that monumental clinical achievements should not overshadow the importance of investing in robust, scalable manufacturing processes and commercial planning a lot earlier in development. ATM's are shifting the nature of drug R&D risk and establishing manufacturing as a critical strategic priority alongside therapeutic safety and efficacy while introducing new commercialization challenges. https://lnkd.in/g2SkUVJH #cellandgenetherapy #biotech #kitepharma #healthcaredesign

We’ve nominated the first candidate from our proprietary pipeline of novel immunometabolism-targeting therapies – the oral, targeted pyruvate kinase M2 (PKM2) activator, SYX-5219 – for clinical development.   SYX-5219 offers first-in-class potential as a disease-modifying, anti-inflammatory therapy for atopic dermatitis, the most common inflammatory skin disease.   Regulatory submission preparation for SYX-5219 is underway, with clinical trials expected to commence in early 2025.   Learn more here: https://lnkd.in/eS8FGNRx   #CellMetabolism #DrugDiscovery #DrugDevelopment #Immunometabolism #Biotechnology #Biotech

Orchard Therapeutics’ ultra-rare disease drug #Lenmeldy is vaulting to the list of most expensive drugs ever with a wholesale acquisition cost of $4.25 million for the one-time treatment in the US.... ....In justifying the price tag, Orchard said that it referenced the Institute for Clinical and Economic Review’s (ICER) assessment, which set the health benefit price benchmark for Lenmeldy at up to $3.94 million.... ...The company added that the price is “reflective of the value the therapy may deliver to eligible patients and their families, as well the potential long-term impact treatment may have on overall healthcare utilization, minimization of productivity loss for caregivers, and life opportunities for patients.”... Bennett Smith Institute for Clinical and Economic Review (ICER) ##onetimetreatment #valueoftherapy #pricing #patientimpact #raredisease #rarediseases #genetherapy #cellandgenetherapy #regenerativemedicine #advancedtherapies #biotech #biotechnology #pharma #pharmaceuticals #lifesciences #healthcare #patientacccess #reimbursement #metachromaticleukodystrophy #MLD #children #ultrararedisease #economicvalue Kyowa Kirin Co., Ltd. #USpricing

Harvard Business Review article today - "Advanced Therapeutic Modalities ...are shifting the nature of drug R&D risk and establishing manufacturing as a critical strategic priority alongside therapeutic safety and efficacy while also introducing new commercialization challenges." Biopharma must focus earlier on ATM manufacturing and supply chain. #celltherapy #genetherapy #celltherapymanufacturing #coldchainlogistics #CARTcellTherapy

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Utilizing animal models for in vivo research in transdermal drug delivery (TDD) presents several drawbacks, including high costs, unreliable predictions of human pharmacokinetics (PK), and ethical concerns within the industry. Furthermore, current computational models for transdermal administration encounter numerous challenges. As the popularity of this route of administration grows, I've noticed an increasing number of inquiries about how we tackle this challenge. Therefore, I thought it would be beneficial to share our approach here. By combining Machine Learning (ML) with mechanistic modeling, our #hybridAI approach enables our #BIOiSIM® platform to accurately predict transdermal drug disposition across various compounds. This approach effectively characterizes both the observed exposure and the intended pharmacological effect. In the image below, I provide detailed insights into how we developed our solution, along with results that demonstrate the predicted drug exposure for three tested compounds – morphine, buprenorphine, and nicotine – achieving an average accuracy of 82%. Our approach to model development is significant because it enables scalable, accurate simulations crucial for evaluating the potential of compounds as transdermal therapeutics. This represents an exciting advancement in in silico prediction as transdermal drug delivery becomes increasingly popular for its painless and noninvasive qualities, and the necessity to lower costs and enhance translatability in optimization methods grows.

BMS-backed liquid biopsy biotech Precede Biosciences debuts to enhance precision medicine approaches >>> lqventures.com #strategy #competitiveintelligence #marketing #pharma #biotech #pharmaceutical #productmarketing #healthcare

Thoughts on this? >> Prime Medicine receives FDA clearance to run first prime editing clinical trial >> Comment below! >>> lqventures.com #strategy #competitiveintelligence #marketing #healthcare #biotech #pharmaceutical #pharma #productmarketing

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