Michael Kahn

FDA expedited designation trends are a marker of innovation, promise, and momentum in the R&D pipeline, particularly for cutting-edge therapies. Earlier this year, Peter Marks, director of the Center for Biologics Evaluation and Research (CBER) declared that “accelerated approvals will be the norm”, especially for initial approvals of gene therapies. Six months later, this trend is indeed accelerating. What’s more, regenerative medicine advanced therapy RMAT requests and designations are emerging as a clear preference to other expedited pathways. Read our quarterly discussion on designation trends for advanced therapies to learn more about the basis for each individual therapy designation as well as commentary and analysis from recognized Parexel experts that offer additional context on data and findings. https://lnkd.in/gxKsPgNC #CBER #Regulatory #RMAT #FDA

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We're thrilled to attend the 2024 International HBV Meeting in Chicago next week! As part of our commitment to advancing research and treatment for viral and life-threatening illnesses, we're hosting a panel discussion on the critical role of patient perspectives in Hepatitis B research and treatment. Anne Kasmar, Parexel's Senior Vice President and Therapeutic Head of Infectious Disease, will moderate the discussion with a panel of experts representing diverse experiences in the Hepatitis B research community: - Patient leader- Wendy Lo, Patient Advocate - Foundation leader- Chari Cohen, President, Hepatitis B Foundation - Industry leader- Christine Livingston, Scientific Director, GlaxoSmithKline - Clinician- Roger Inouye, Senior Medical Director, Parexel - Academic researcher- Thomas Tu, Group Leader, Storr Liver Centre, The Westmead Institute Together, we'll explore how patient insights drive innovation, improve trial design, and shape research priorities in the fight against Hepatitis B. We'll discuss current challenges in Hepatitis B research and treatment and how increased patient engagement and collaboration can help overcome these obstacles. We look forward to a transformative discussion highlighting how patients will reshape the future of Hepatitis B research and treatment. Let's embrace a patient-guided approach across all phases of research to propel progress in treating and finding the cure for Hepatitis B! https://lnkd.in/gaSDzXCX #HepatitisB #HepB #PatientPerspectives #PatientFirst

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The Chamber of Commerce for Greater Philadelphia released “Leading the Way: Cell and Gene Therapy in Greater Philadelphia Sector Snapshot 2024." This in-depth report describes the Greater Philadelphia region’s remarkable growth journey in the cell and gene therapy (CGT) sector over the past decade, showcases how Greater Philadelphia has emerged as a global leader in CGT and highlights a variety of Penn’s contributions to the field. The report notes that Penn has spun out 40 companies since 2018, which has significantly contributed to the growth of the CGT sector in Greater Philadelphia. In addition, Penn Medicine was recognized as a top location for Phase I and II trials in novel cell and gene therapies and the Penn mRNA Institute recently received a $350 million investment. All of this has helped further grow the expanding CGT space in Philadelphia, which has received over $8 billion in private capital invested since 2018 and generated 7,000 jobs. Read the full report here: https://lnkd.in/g-UKX6Rc 

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Roche Doubles Down on Breast Cancer With $850M for Regor’s CDK Inhibitors - BioSpace The acquisition was featured Monday in Roche’s Pharma Day presentation, which also included projections of more than $3 billion in annual sales from three early-stage obesity and diabetes drugs. A portfolio of CDK inhibitors developed by Regor Pharmaceuticals has found a home at Roche, which announced Monday it would buy the investigational cancer drugs for $850 million upfront. Cambridge, Mass.– and China-based Regor will see two ongoing Phase I trials of CDK4 inhibitor RGT-419B through to their completion, at which time Roche’s Genentech subsidiary will take over global clinical development, manufacturing and commercialization of this asset. The deal also includes additional CDK4 inhibitors for cancer. Last December, Regor announced results from a Phase Ia trial of RGT-419B, showing that the drug was safe and well tolerated in patients with HR /HER2- advanced breast cancer who have progressed on CDK4/6 inhibitors and endocrine therapy. “Monotherapy clinical efficacy” was also observed, Regor reported. The deal, which is expected to close in the fourth quarter of this year, includes the potential for Regor to claim additional cash payments based on the achievement of predetermined development, regulatory and commercial milestones. “Genentech is well-positioned to bring these novel therapeutics to their full potential to benefit patients with breast cancer around the world,” Xiayang Qiu, founder and CEO of Regor, said in a statement. https://lnkd.in/eU-gY4wQ

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Looking forward to conversations around therapies and technology progressing with improved investor sentiment, albeit with caution! Investing? How commercially viable is the science? What are the product development/CMC and regulatory/clinical risks? What is the competitive landscape? Fundraising? Does your pitch deck and data room position your company to be as investable as possible? Promising non-clinical data? How to turn that into a commercial product? What is the best product development and regulatory strategy? Growing pains? Addressing the various resource gaps in regulatory affairs, regulatory operations, clinical operations, clinical and medical affairs, regulatory and medical writing and many more as you transition the product from one phase to another! Let’s connect and discuss how Boyds can help! #REAIBoston2024 #BiotechweekBoston

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