Timothy Schroeder

Really interesting news from the last month: Blackstone, the parent company of Advarra IRB and Precision For Medicine CRO, just announced a $750MM direct investment into Merck’s Oncology compound sac-TMT (sacituzumab tirumotecan). https://lnkd.in/eMgrN4K7   Understanding Blackstone owns Advarra and Precision, and given the most recent The New York Times article on potential conflicts on interest in clinical research, this deal seems to land squarely in the middle of the current conversation about real and perceived conflicts of interest in clinical research oversight. It also raises fair questions about which CRO and IRB partners Merck may feel comfortable engaging for sac-TMT trials, and how sponsors should think about independence when investment/ownership ties are aligned within multiple aspects risking independence of work.    Brad Hightower, Denali Rose, walt bogdanich (and others) – this seems rather timely based on your recent Note to File: A Clinical Research Podcast What are your thoughts?

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Today marks an important milestone for MiNK Therapeutics and for patients undergoing allogeneic stem cell transplantation. We are proud to announce our clinical trial of agenT-797, our allo-INKTs, for the prevention of graft-versus-host disease (GvHD). This non-dilutive, public-private partnership, supported by National Institute of Allergy and Infectious Diseases (NIAID) STTR award and the Mary Gooze Clinical Trial & Translation Award, in collaboration with the University of Wisconsin-Madison, allows us to advance translational science and clinical execution in parallel—exactly how innovation should move. Why this matters: • GvHD remains one of the most serious and persistent complications of allogeneic HSCT • iNKT cells uniquely suppress harmful allo-immune inflammation while preserving anti-leukemia immunity • agenT-797 is HLA-independent, lymphodepletion-free, and off-the-shelf, with prior clinical safety across oncology and severe pulmonary inflammation Our goal is to reduce GvHD and relapse while supporting immune reconstitution, without adding cytotoxic burden for patients already fighting for recovery. Thank you Dr. Hongtao Liu, Dr. Kalyan Nadiminti, Dr. Jenny Gumperz, and the UW–Madison team for their leadership and partnership. This program reflects years of shared translational work and a deep commitment to improving post-transplant outcomes. At MiNK, we believe immune reconstitution is the next frontier of cellular therapy—and this study is a meaningful step forward. 🔗 Read the full announcement: https://lnkd.in/euuXGFay #MiNKTherapeutics #iNKT #CellTherapy #GvHD #StemCellTransplant #Immunotherapy #PublicPrivatePartnership #LivingMedicine

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Not all FDA pathways are the same. When it comes to chronic venous leg ulcers (VLU), the regulatory standard matters. Premarket Approval (PMA) requires: • Prospective, randomized clinical trials • Pre-specified endpoints • Statistical powering • Demonstrated safety and effectiveness Currently, Apligraf® is the only therapy approved through the FDA PMA pathway for the treatment of chronic venous leg ulcers. In a category often defined by equivalence or post-market data, that level of evidence stands apart. For patients. For providers. For payers. Because when healing is the goal, proof matters. Organogenesis #woundscantwait Apligraf.com

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👩‍🔬💬 𝐂𝐞𝐥𝐥 & 𝐆𝐞𝐧𝐞 𝐓𝐡𝐞𝐫𝐚𝐩𝐲 𝐏𝐨𝐬𝐢𝐭𝐢𝐯𝐢𝐭𝐲: 𝟐𝟒𝐭𝐡 𝐅𝐞𝐛𝐫𝐮𝐚𝐫𝐲 𝟐𝟎𝟐𝟔 👨‍🔬💬 🦠 Gilead Sciences is acquiring Arcellx for $7.8 billion to take full ownership of anitocabtagene autoleucel (anito‑cel), a BCMA‑directed CAR‑T therapy nearing FDA approval for fourth-line multiple myeloma. Clinical data show deep, durable responses and a manageable safety profile. Gaining full ownership removes profit‑sharing and royalty obligations and is expected to accelerate development and enable broader commercialization. 🧬 Ultragenyx announced that the FDA has accepted and granted Priority Review for its BLA seeking approval of DTX401 (pariglasgene brecaparvovec), an AAV8 gene therapy for Glycogen Storage Disease Type Ia. The PDUFA date is set for August 23, 2026. If approved, DTX401 would be the first therapy to address the genetic root cause of GSDIa, reducing reliance on burdensome cornstarch‑based dietary management. 🦠 Zelluna has received MHRA and Research Ethics Committee approval to begin the first‑in‑human Phase I trial of ZI‑MA4‑1, its lead TCR‑NK cell therapy. The study marks Zelluna’s transition into clinical‑stage development. Initial data are expected in mid‑2026. The Phase I trial will evaluate safety, tolerability, and preliminary activity in patients with advanced solid tumours, including lung, head and neck, and ovarian cancers. 🧬 Affinia Therapeutics has received EMA Orphan Drug designation for AFTX‑201, an investigational AAV gene therapy for BAG3‑associated dilated cardiomyopathy (DCM), a severe inherited heart disease with high mortality and limited treatment options. AFTX‑201 delivers a full‑length BAG3 transgene via a proprietary cardiac-targeted capsid engineered to enable effective gene delivery at significantly lower doses than conventional AAV vectors. 🧬 Tessera Therapeutics has received FDA Fast Track and Orphan Drug designations for TSRA‑196, its lead in vivo Gene Writing therapy for adults with alpha‑1 antitrypsin deficiency (AATD) caused by the PiZZ genotype. Developed with Regeneron, TSRA‑196 aims to permanently correct the underlying AATD mutation through a one‑time treatment that restores functional AAT production. 🧬 YolTech Therapeutics reported positive interim data from a first‑in‑human study of YOLT‑202, its in vivo base‑editing therapy for alpha‑1 antitrypsin deficiency (AATD). Two PiZZ patients treated at 35 mg and 45 mg showed rapid, dose‑dependent and clinically meaningful increases in functional AAT levels. Over 95% of produced protein was corrected M‑AAT. YOLT‑202 demonstrated a favourable safety profile, with only mild, manageable adverse events. 🛎 My aim is to be the messenger when it comes to your CGT & RNA therapy news. Follow #mCGT to stay updated! #celltherapy #genetherapy #ATMP #advancedtherapy

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Top 10 CVRM Takeaways From JPM 2016 (Day 1)- Part II Ike Ogbaa, MD Iroko Lifesciences Consulting 6. Commercial execution is becoming as important as clinical data Dexcom, Cytokinetics, and Madrigal demonstrated that: • OTC/consumer channels (CGM, obesity) • Focused specialty launches (HCM, MASH) • App ecosystems and adherence tools are now core CVRM success factors, not “nice to haves.” ⸻ 7. Cash-pay dynamics are reshaping CVRM launch strategy Pfizer explicitly acknowledged that ~30% of obesity sales are cash-pay, reframing payer-first thinking. This is pushing companies to: • Accelerate launch timelines • Emphasize consumer experience • Build brand trust earlier than traditional CV drugs ⸻ 8. MASH remains a crowded but still open field Madrigal Pharmaceuticals remains first-mover, but JPM reinforced that: • MASH is biologically multi-factorial • Combination regimens (thyroid hormone + DGAT2 + incretin) are inevitable • Diagnosis, not just drug efficacy, is the near-term bottleneck ⸻ 9. Precision cardiometabolic medicine is emerging Arrowhead’s comments on obesity subtypes, Novartis’ Lp(a) focus, and targeted RNAi approaches reflect a pivot away from “one-size-fits-all CVRM.” Future programs will increasingly stratify by: • Genetics • Inflammatory phenotype • Lipid patterns • Organ-specific risk ⸻ 10. The bar for “platform value” has risen significantly Across JPM, platforms that clearly demonstrated: • Multiple shots on goal • Cross-indication scalability • Lifecycle extension pathways were rewarded with stronger narratives. Incremental assets without a portfolio-level story struggled to stand out. ⸻ Bottom Line for CVRM Leaders JPM 2026 made one thing clear: CVRM is converging into integrated cardiometabolic platforms, where obesity, lipids, inflammation, diabetes, and CV outcomes are treated as one continuum, not separate silos.

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HEMORRHOIDS: OVERVIEW #Hemorrhoids, which are vascular and connective tissue structures lining the anal canal, are present in all individuals, although the term is commonly used to refer to a diseased state. The symptomatic, pathological condition that requires medical attention is better described as hemorrhoidal disease. Although not a life-threatening condition, #hemorrhoidal_disease reduces quality of life due to anal bleeding, pruritus, rectal pain and pressure, and sensation of tissue prolapse. Hemorrhoids are classified as internal, external, or mixed (concurrent internal and external hemorrhoidal disease). Internal hemorrhoids originate above the dentate line, the boundary between the upper and lower anal canal, and may cause rectal bleeding, discomfort, and tissue prolapse from the anal canal. Internal hemorrhoid prolapse is classified as grade I (into anal canal), grade II (beyond the anus with spontaneous reduction), grade III (requiring manual reduction), and grade IV (irreducible). External hemorrhoids, arising below the dentate line, cause rectal pain when engorged or thrombosed. Treatment decisions are based on symptom severity, and asymptomatic hemorrhoidal disease does not require treatment. Initial treatment of all hemorrhoidal disease involves increasing intake of dietary fiber and water and avoiding straining during defecation. Phlebotonics (eg, #flavonoids) reduce bleeding, rectal pain, and swelling, although symptom recurrence reaches 80% within 3 to 6 months after treatment cessation. This review - open access on JAMA - summarizes current evidence on the epidemiology, pathophysiology, diagnosis, and treatment of hemorrhoidal disease. Ashburn JH. Hemorrhoidal Disease: A Review.  JAMA, August 18, 2025. doi:10.1001/jama.2025.13083 https://lnkd.in/d-a6e4Kn

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📢 Regulatory Update – VYJUVEK® (beremagene geperpavec-svdt) The FDA has approved an expanded label for Krystal Biotech’s VYJUVEK, the first and only topical gene therapy for dystrophic epidermolysis bullosa (DEB). Key updates include: 🔹 Patient eligibility from birth – extending access to the most vulnerable patient population 🔹 Self- or caregiver application – enabling administration outside healthcare facilities, including at home 🔹 Revised wound dressing guidance – dressings may now be changed at the next routine schedule, instead of the prior 24-hour restriction These changes are supported by real-world evidence and open-label extension studies, reinforcing both the safety and efficacy profile across all age groups. Why it matters: Earlier intervention may mitigate disease burden from infancy Decentralized administration improves accessibility and quality of life Alignment with standard wound care practices optimizes patient management This update underscores the evolving role of gene therapies in rare dermatological disorders and highlights the importance of regulatory flexibility informed by real-world data. #GeneTherapy #RareDisease #RegulatoryAffairs #Dermatology #ClinicalResearch

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