Rodney Marable

Demonstrating the technical leadership of SK pharmteco! 🧬 Catch up with our team at ASGCT to discuss how SKPT can support your #cellandgene development!

9

1 Comment

Umoja Biopharma Announces Publication in Blood Highlighting Successful Generation of In Vivo CAR T-cells in Non-Human Primate Modeling First landmark, peer-reviewed publication of successful in vivo CAR T-cell data in an immune competent NHP model Data validate the VivoVec™ platform in a translationally relevant model and support its transition into human clinical testing, offering a potential paradigm shift in the field of CAR T-cell therapies Key Study Findings VivoVec particles (VVPs) incorporating CD80 and CD58 costimulation proteins exhibit enhanced capacity for in vivo CAR T-cell generation and produce CAR T-cells with increased antitumor functionality compared to those produced from VVPs displaying anti-CD3 scFvs alone. Combining the anti-CD3 scFv together with the ligand binding domains of CD80 and CD58 into a single multi-domain fusion (MDF) protein markedly augments the particles’ ability to bind, activate, and transduce T-cells. VVPs incorporating MDF compatible with NHP T-cell activation and costimulation potently generate anti-CD20 CAR T-cells in vivo, comprising up to 65% https://lnkd.in/eqsSTKjf

18

🔬 Discovering Biomarkers for Better Outcomes in Melanoma Treatment 🔬 Exciting news from ASCO 2024! Led by Jeffrey Weber, our team is proud to present pioneering findings on the role of predictive and pharmacodynamic biomarkers for response and toxicity in melanoma treatment. Our phase II trial combined tocilizumab (TOCI) with immune checkpoint inhibitors, reducing toxicity and revealing new biomarkers. Initiated in 2020-21, this work involved multiple high parametric flow cytometry panels to identify various myeloid, NK and functional CD4 and CD8 T cell populations linked to treatment outcomes. Our insights are paving the way for more personalized, effective melanoma management. 🔥 Hot Take: Inflammation Modulators are Here to Stay in Immuno-Oncology!🔥 📍 Check out our findings at ASCO Poster Abstract #9538; Poster Board #322. #ASCO2024 #MelanomaAwareness #TranslationalBiomarkers #TranslationalResearch

28

OPCU - Partner Week: Biotech's developing large molecule drugs should attend, finding the right outsourcing partner is critical to drug development strategy! At OPCU events, get your questions answered and find out what you need to know within your selection process of working with CDMO partners, timing , capabilities and cultural fit can all be assessed at the Outsourced Pharma Capacity Update. Just Ask for a follow up meeting after the presentation!

3

📣Call all RNA-targeting experts📣 The 2nd RNA Assay Development & Screening Summit is returning to Boston this August to accelerate RNA-targeted drug discovery! This meeting will enable you to: 🔴 Expand your biophysical screening strategies to better understand the interactions between your small molecule drugs and RNA targets 🔴 Discover innovative assay strategies used for understanding the true mechanism of action of small molecules targeting RNA 🔴 Identify selective RNA binders through robust and reproducible assay and screening strategies to fast-track more effective drugs towards the clinic 🔴 Optimize tools for understanding the RNA target structure to improve binding and druggability Not to mention experts from Arrakis Therapeutics, Atavistik Bio, Wayfinder Biosciences, Biogen, ReviR Therapeutics, Accent Therapeutics, Inc. and many more will all be sharing BRAND-NEW DATA! Check out the full event guide here: https://ter.li/z3ea9l P.S. This Friday, June 7, is the deadline to save up to $800 on your registration pass for this unmissable meeting. #smallmolecules #RNA #RNAtarget #RNAtargeting #splicing #longnoncodingRNAs #RNAbindingproteins

8

It’s difficult to navigate the balance of phase-appropriate needs with commercial readiness during cell and gene therapy development. Partnering with a trusted and experienced viral vector #CDMO can help unlock unique insights and provide guidance to achieve the right balance for your therapeutic. Here are 4 recommendations to keep in mind during preclinical and clinical development:

1

BDRA Consulting LLC has been a member of AMDM since 2013. Come join AMDM for the entire story. Twice a year, members get to meet with the FDA to discuss upcoming regulatory and quality policies. The FDA provides industry updates. This year's meeting was held at Building 66, White Oak campus on May 21, 2024. Key Takeaways from the May 2024 AMDM Industry FDA Roundtable: 1. Cybersecurity for medical devices is a key issue. OHT7 and CDRH have increased focus on cybersecurity for medical devices. 2. The Oncology Diagnostic Pilot Program is nearly a year old, and no company has been accepted. This is primarily because drug manufacturers have not been able to provide validation data. 3. Reclassification plans for companion diagnostics are ongoing and will be completed by November 2027. - FDA is considering down-classifying three types of CDxʼs: i. Nucleic acid-based CDX ii. In situ hybridization-based CDX iii. Immuno-histochemistry-based CDX - Technologies will be grouped based on the intended use. 4. PCCP guidance for medical devices will be published before the end of the fiscal year 2024. A PCCP may be used to make changes to sample type changes, which may be acceptable in certain cases. 5. QSIT is being replaced by an entirely novel risk-based approach, which is currently being worked out. Manufacturers should be working through transition plans. #AMDM

11

Want to know how AstraZeneca's Irina Ramos and AbbVie's Sanjay Nilapwar are intensifying #biopharma processes and mapping their next #continuousmanufacturing steps? Then ask them! This talk, 6/11 at 11 AM ET, promises to be a good one, and it's less than a week away. Register here, at no charge, and get in on the conversation! https://lnkd.in/eZ4-WiJq

2

Have you thought about health equity and gene therapy? Gene therapies are expensive! Pfizer’s newest hemophilia gene therapy is peaking at 3.5 million dollars a treatment. Talk about sticker shock. This is not accessible for many and for the “traditionally underserved and marginalized groups” this is nearly impossible. Mary A. Majumder and Titilope Fasipe write a cogent and timely essay in Ethics and Human Research Vol. 46, Issue 3 based on the National Academies of Science, Engineering, and Medicine report, on achieving fair and equal health innovations for everyone, titled “Toward Equitable Innovation in Health and Medicine: A Framework.” Majumder and Fasipe focused on two key areas of that report in their essay: First, input equity - making sure the voices of communities impacted by health issues are included in the research and development process. No more excluding the people it aims to help. Second, deployment equity - ensuring these new innovations actually benefit diverse populations, especially groups that are underserved or marginalized. Not just advantages for the usual populations. The report uses the approval of two new gene therapies for sickle cell disease in December 2023 as an example. While these therapies are major breakthroughs, they are very expensive ($2.2-$3.1 million per patient) and complex to administer. This raises concerns that they may not be equally accessible, especially for underserved populations most impacted by sickle cell disease. The government tried providing incentives like extended patents to get private companies to invest in developing the therapies. But there weren't any requirements around pricing to promote affordability. Some efforts were made to gather community perspectives and analyze costs to keep things equitable, but obstacles remain. Issues like lack of insurance coverage, doctor shortages, and mistrust in the healthcare system stand in the way of these new gene therapies reaching many sickle cell patients, at least in the short term. This example shows how important research funders are for promoting fairness, and the need for a comprehensive approach that considers real-world healthcare experiences and costs. The key lesson is that research funders have a crucial role to play in promoting true equity. And we need a comprehensive approach that deeply understands the real-world healthcare experiences and economic challenges people face. Cracking this is vital for ensuring all groups benefit from major health innovations. #HealthEquity #SickleCellDisease #GeneTherapy #HealthPolicy Links in the comments.

14

3 Comments

Don't miss it. 📆 Matthew Pillar's talking to two career pros in #SingleUse systems, Mark Petrich and Paul Priebe, during a Bioprocess Online Live event on April 24. Here's a peek at their agenda for the day. 🍃 Sustainability — despite the extra plastic waste, evidence shows #SUT is more sustainable. 💸 Costs — over time, SUT can cost 25% less than stainless. ⚖ Standards — SUT are pretty well established, but still evolving. ⛓ Supply chain — interoperability remains a big watch-out as more SUT manufacturers enter the field.

12

Genentech Wins FDA Approval for First Adjuvant Therapy for ALK-Positive, Early NSCLC The FDA on Thursday greenlit the use of Genentech’s Alecensa (alectinib) for the adjuvant treatment of patients with early-stage, anaplastic lymphoma kinase-positive non-small cell lung cancer who have undergone tumor resection. With the approval, Alecensa is now the “first and only” ALK inhibitor approved for this indication and patient population, according to Genentech. Thursday’s regulatory win is backed by data from the Phase III ALINA trial, a randomized, active-controlled and open-label study comparing adjuvant Alecensa with platinum-based chemotherapy. The study enrolled 257 patients with Stage IB to IIIA tumors who had undergone resection and tested positive for anaplastic lymphoma kinase (ALK) using an FDA-approved test. Results from ALINA, which the company released in October 2023, showed that Alecensa could cut the risk of disease recurrence or death by 76% versus platinum-based chemotherapy. This treatment effect was highly significant and yielded a p-value of less than 0.0001. The ALK blocker also led to a significant improvement in central nervous system-disease-free survival, according to an exploratory analysis of ALINA. Genentech CMO Levi Garraway in a statement on Thursday called ALINA’s disease-free survival data “unprecedented,” adding that “Alecensa significantly improves upon the standard of care for people with early-stage ALK-positive lung cancer.”

88

1 Comment

Regeneron's blockbuster eye drug posts weaker sales due to inventory impact May 2 (Reuters) - U.S. drugmaker Regeneron Pharmaceuticals Inc (REGN.O), opens new tab missed Wall Street estimates for quarterly profit on Thursday, hurt by softer sales of its blockbuster eye drug Eylea due to reduction in wholesaler inventory. Apart from an impact of about $40 million cut in inventory, Regeneron said changing market dynamics that resulted in lower volumes and lower selling price also hurt Eylea sales. Total sales of the drug, jointly developed with Bayer AG (BAYGn.DE), opens new tab, fell 2% to $1.40 billion in the January-to-March quarter, missing estimates of $1.86 billion, according to LSEG estimates. The higher 8 milligram dose version contributed $200 million to Eylea's total sales, compared to consensus estimates of $196 million. Regeneron said it continues to see strong uptake of the higher dose version of Eylea. Analysts have high hopes for the high dose. They expect the high-dose version to gain market share over the standard dose largely over the next year. https://lnkd.in/eZ-NqKDH

4

Exciting data at #ASCO24 for a combination regimen including our ADC demonstrating clinically meaningful improvement in overall survival among patients with relapsed or refractory diffuse large B-cell lymphoma (#DLBCL). Learn more: https://lnkd.in/guwDmfzG   This is the third Phase 3 study in a type of lymphoma to demonstrate overall survival benefit for a combination regimen containing our ADC. These data add to the growing body of evidence, and subject to regulatory review, may help further evolve the standard of care in multiple types of lymphoma. 

184

1 Comment

Learn from our team how LC-MS option can save months off your timeline

7

Appreciate John Glasspool’s op/ed in the Boston Business Journal. He brings the perspective of a CEO of a Cambridge-based #biotech, MassBio member Anthos Therapeutics, to the debate over the White House’s plans to use Bayh-Dole as a blunt instrument to force price controls. The reality is that any effort to use “march in” rights like this would hurt innovation, chill the collaborative relationship between research institutes and small biotechs (which are the lion’s share of Massachusetts’ industry) when it comes to advancing early science, and do nothing to address the price of drugs. You can’t argue with Bayh-Dole’s success: 6 million jobs, $2 trillion in economic output, thousands of startups, and 200+ drugs and vaccines. As John points out, we can’t go back to a time when science sat on shelves instead of getting into medicine cabinets. https://lnkd.in/ezkNndKA cc: The Bayh-Dole Coalition

32

3 Comments

Jonathan Montagu, CEO of MassBio member HotSpot Therapeutics, Inc., writes on the #LifeSCiVC blog about hopes around an immuno-oncology spring this year, aligning with the emergence of #biotech from its own long winter. Jonathan points out that after the #lifesciences industry dove headfirst into I-O “to address the substantial need that still persists for many cancer patients with a range of tumor types,” much of the second-generation agents fell short. He writes about why he’s optimistic that new approaches have started to turn the tide. What are your thoughts on the I-O space right now? https://lnkd.in/eMh62Z8j #immunooncology #oncology #patientdriven

28

We started out Q1 with cautious optimism and the numbers continue to show the confidence was warranted. Biopharma M&A activity more than doubled nationally during the year’s first three months, coinciding with a rebound in venture capital deals as well as a flurry of IPOs. These are all good signs that the drug development cycle is normalizing and should translate into additional funding being available at the seed and Series A stages. Certain to be on the minds of funders and startups attending #AlignSummit24 on Thursday! https://lnkd.in/erEiJm6S  

112

5 Comments