Richard Zwickel

Roche on Thursday inked its second research collaboration deal with Dyno Therapeutics to advance next-generation gene therapies for neurological diseases. Under the terms of the agreement, Roche will make an upfront payment of $50 million and pledge more than $1 billion in preclinical, clinical and commercial milestones, plus royalties on net sales of any product that arises from the partnership. Roche will gain even greater access to Dyno’s in vivo gene therapy delivery platform, which uses AI to optimally and rapidly synthesize adeno-associated virus (AAV) capsids. According to the biotech’s website, this approach can sidestep the common limitations of naturally occurring capsids—which existing gene therapies use—such as problems with existing immunity, payload size and manufacturability. #biotech #biopharma #pharma #genetherapy

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Exciting news in biotech - Q1 2024 saw a 46% surge in venture funding, signaling a strong recovery. Late-stage clinical developments and innovative therapies like ADCs and radiopharmaceuticals received significant investments. The important question is what does this mean for the future of biotech? How will these trends impact the development of new therapies? And how is investor confidence changing as the IPO market sort of re-opens? Interesting thoughts to consider. You can read more on our blog: https://ow.ly/bpCF50S2iY6 #Biotech #VentureCapital #HealthcareInvestments

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The collaboration between Virpax Pharmaceuticals Inc. (Nasdaq:VRPX) Pharmaceuticals and the National Center for Advancing Translational Sciences (NCATS) heralds a significant advancement in pain management research. Focused on the development of NES100, an intranasal peptide targeting acute and chronic non-cancer pain, this partnership utilizes innovative nanotechnology to enhance the delivery of enkephalin to the brain. By leveraging Molecular Envelope Technology (MET) and a specialized device, NES100 aims to provide analgesic effects without the adverse events commonly associated with opioids. This extended collaboration underscores the shift towards non-opioid alternatives for pain management, crucial in addressing the opioid crisis. The research aligns with the NIH’s HEAL initiative, emphasizing the national commitment to combat opioid addiction. The potential of NES100 lies in offering a non-addictive pain treatment, potentially transforming pain therapy and improving outcomes for individuals with chronic pain conditions. NES100's development presents a promising avenue for pain management, offering a safer alternative to traditional opioids. If proven effective in clinical trials, NES100 could emerge as a valuable tool for patients seeking pain relief. The intranasal delivery method not only enhances convenience but also holds the promise of improved patient adherence and treatment efficacy. 𝐑𝐞𝐚𝐝 𝐌𝐨𝐫𝐞: https://lnkd.in/dwMpJZf8 Gerald W. Bruce Matt Barnes

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With 15 patients started across its three gene therapies, bluebird bio claims a nearly 140% year-over-year revenue growth and aims to initiate up to around 100 new patients in the current year. The Massachusetts-based biotech revealed that it has so far initiated 15 patients on its gene therapies, one of whom started the recently approved sickle cell disease treatment Lyfgenia just this month. The remaining 14 patients are receiving either Zynteglo, for pediatric beta-thalassemia, or Skysona, which is intended for cerebral adrenoleukodystrophy in boys. William Blair analyst Sami Corwin, Ph.D. anticipates that Lyfgenia will face stiff competition from CRISPR Therapeutics and Vertex Pharmaceuticals’ Casgevy, which the analyst predicts will be a “headwind” for bluebird. One key advantage for Casgevy is its price. Bluebird gave Lyfgenia a price tag of $3.1 million, nearly $1 million more than Casgevy’s $2.2 million. #biotech #biopharma #sicklecelldisease #genetherapy

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Fast track for FDA by June 30 will release its verdict on #Rocket Pharmaceuticals’ BLA for #Kresladi (marnetegragene autotemcel), an investigational #gene therapy the New Jersey-based biotech is proposing as a treatment for severe #leukocyte adhesion deficiency-1 (LAD-1). Severe LAD-1 is a rare #pediatric disorder caused by mutations in the #ITGB2 gene, which encodes for a crucial component of the CD18 protein. Under healthy circumstances, #CD18 helps leukocytes exit blood vessels to combat infections. In severe #LAD-1, which typically manifests during early childhood, patients suffer from recurrent and unresponsive bacterial and fungal #infections.

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In a recent interview, Alisha Garibaldi, CEO of Skylight Health Research, Inc, shared valuable insights on managing clinical research sites and the strategic use of technology to enhance efficiency. Garibaldi emphasized the importance of gradual tech adoption, standardization, and clear communication with sponsors. She highlighted Skylight’s approach to integrating CTMS and leveraging platforms like Veeva Site Vault to streamline operations. For sites and sponsors, collaboration and transparency remain key. #ClinicalResearch #CTMS #SiteManagement #Veeva #ClinicalTrials Deivis Mercado Veeva Systems

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#ICYMI Last year, MedImpact clients achieved higher Star Ratings on average than those of any of the three largest PBMs in key medication adherence categories: Diabetes, Hypertension, Cholesterol, and Statin Use in Persons with Diabetes. Ask us how. https://bit.ly/3jlUw1s #wearemedimpact #atruepartner #healthcare #pharmacybenefits #CMSStarRatings

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“Scripius worked with one of its clients, Select Health, to quickly transition 98% of their Humira prescriptions to one of the adalimumab biosimilars. We believe this was the fastest transition in the country. “ Samsung Bioepis, recently sat down with our Pharmacy Services AVP, Matt Mitchell, to pick his brain on biosimilars' strategy. Matt explains the financial calculus, the obstacles of communicating a change to diverse audiences, and what he has learned from Scripius’ most recent biosimilars move. Here's the Q&A - https://lnkd.in/gd7SABgA #PBM #PharmacyBenefits #Biosimilars

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The number of biopharma M&A deals more than doubled in the first quarter of 2024 compared to the same period a year ago, exemplifying a top exit route for pre-commercial companies vying for validation and attention. 13 biopharma M&A deals in the first quarter of 2024 compared to six in the first quarter of 2023. 47 deals in the last twelve months through March 2024, compared to 42 through March 2023. #mergerandacquisition #investing #CEO #biopharma #wheretoinvest #druginnovation #artificialinyelligence

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The Medicine Maker feature article - Don’t Forget the Elderly Sharmeen Roy, PharmD, BCPS, Chief Strategy and Science Officer at DoseMe, discusses how across the board, older adults are significantly underrepresented in clinical trials. In fact, they comprise around 25–40 percent of clinical trial participants, despite making up the majority of prescription drug users and having the highest disease burden. Read the full article that dives into pharmacokinetic differences, safety endpoints and efficacy analysis. https://lnkd.in/eUxbabdq #pharmacokinetic #safety #precisiondosing

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In a late-stage trial, Insmed’s oral small molecule DPP1 inhibitor cut yearly exacerbation rates by around 20% in bronchiectasis patients, according to topline data. The biotech's shares soared more than 100% in reaction to the news. The biotech will continue analyzing data from ASPEN and will build toward a regulatory submission for brensocatib, which it plans to submit in the fourth quarter of 2024. The U.S. product launch is slated for mid-2025, pending approval. Designed to be taken orally, brensocatib is a small molecule inhibitor of DPP1, an enzyme that activates neutrophil serine proteases triggering lung damage and inflammation in various chronic immune-driven lung diseases. In addition to bronchiectasis, Insmed is also developing brensocatib for chronic rhinosinusitis without nasal polyps. #biotech #biopharma #bronchiectasis

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A recent analysis from Biomedtracker of clinical trial phase transitions between 2014 and 2023 suggests biopharma development success rates are falling. This poses several questions: what are the underlying reasons dragging success rates down and what does this mean for R&D productivity and the return on investment on pipeline spend? https://lnkd.in/ezX4Dsmb Citeline Commercial #biopharma #clinicaltrials #RnD

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Do you or someone you love suffer from Bipolar Disorder? Alzamend Neuro has partnered with Massachusetts General Hospital for a phase II clinical trial of AL001, a next-generation lithium therapeutic drug candidate, involving patients with Bipolar Disorder. The goal of this clinical trial is to obtain a better biodistribution of lithium in the brain vs. the marketed lithium carbonate in a head-to-head study. This data will be quantifiable via brain scans. Learn more about Alzamends current clinical trials at alzamend.com

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MMIT experts will be at #ASCO24 to discuss why the right tools and real-world data can help uncover insights into the #oncology patient journey. Book a meeting with MMIT: https://ow.ly/zo1I50Ryx1W.

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This week, Mark Campbell, RxBenefits, Inc.'s Chief Pharmacy Officer shared his perspective on the way drug manufacturers, formulary practices by the leading pharmacy benefit managers, and the economics of rebates have led to patients, plan sponsors, and our healthcare system missing out on the significant savings #biosimilars could have delivered. Great insights and lessons we can learn for the future

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Amylyx Pharmaceuticals has acquired avexitide, an investigational therapy for hyperinsulinemic hypoglycemia, from Eiger BioPharmaceuticals. Avexitide, a novel GLP-1 receptor antagonist, has potential applications in treating post-bariatric hypoglycemia (PBH) and congenital hyperinsulinism (HI). The U.S. FDA has granted avexitide Breakthrough Therapy Designation for PBH and congenital HI, along with an Orphan Drug Designation for treating hyperinsulinemic hypoglycemia. Phase 2 studies have shown promising results in reducing hypoglycemic events, paving the way for a Phase 3 trial in PBH in 2025. Amylyx's acquisition underscores its commitment to advancing endocrine and neuroscience treatments for unmet medical needs. Learn more: https://lnkd.in/dK4xSW_r Ryan Miller Tammy Sarnelli Machelle Manuel

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#ICYMI for #Investors and #Media: We shared Phase 1 data in ovarian cancer, and insights into the association of T cell fitness with ImmTAC clinical activity: https://lnkd.in/e9hQ9ga3 #OvarianCancerAwarenessMonth #GCAM

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