"I am pleased to join the Board of TransCode as the company enters an exciting stage of development. I look forward to helping advance our mission to deliver a cancer-free future for patients," said Mr. Stover. Forward-Looking Statements This release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including, without limitation, statements concerning TransCode's technology, collaborations, and team, and statements concerning the therapeutic po ...

LEIDEN, Netherlands & CAMBRIDGE, Mass., Dec. 01, 2025 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies based on its proprietary ADAR-mediated Axiomer™ RNA editing technology platform, today announced that the Company will participate in a fireside chat at the 8th Annual Evercore Healthcare Conference on Thursday, December 4, 2025 at 8:20am ET. Webcast details will be accessible from the “Investors & Media” section ...

Core Insights - Bristol Myers (BMY) is set to acquire Orbital Therapeutics for $1.5 billion in cash, enhancing its pipeline with OTX-201, a next-generation CAR T-cell therapy aimed at autoimmune diseases [1][11] - The acquisition will also incorporate Orbital's proprietary RNA platform, which focuses on durable and programmable RNA therapies [3][11] Company Pipeline and Products - OTX-201 is an investigational in vivo CAR T-cell therapy that utilizes the patient's own body to produce CAR T-cells, potentially reducing treatment burden and improving accessibility compared to traditional ex vivo therapies [2] - BMY's current cell therapy portfolio includes Breyanzi, a CD19-directed CAR T-cell therapy, which has shown significant sales growth of 125% to $344 million in Q2 2025 [4][6] - Breyanzi is approved for multiple indications, including relapsed or refractory large B-cell lymphoma and chronic lymphocytic leukemia [5] Competitive Landscape - BMY's cell therapies face competition from Gilead Sciences' Yescarta and Novartis' Kymriah, which are also approved for similar indications [7][9] - Yescarta reported sales of $393 million in Q2 2025, indicating strong market competition [9] Financial Performance and Valuation - BMY's shares have declined by 18.4% year-to-date, contrasting with the industry growth of 9.2% [12] - The company is trading at a price/earnings ratio of 7.19X forward earnings, which is below its historical mean of 8.45X and the large-cap pharma industry's average of 15.70X [13] - The bottom-line estimate for 2025 has increased slightly to $6.48, while the estimate for 2026 has decreased to $6.02 [14]

Core Insights - ProQR Therapeutics is focused on developing transformative RNA therapies using its proprietary Axiomer™ RNA editing technology platform [1][3] - The company will participate in several upcoming investor conferences, providing opportunities for engagement with investors [1][5] Company Overview - ProQR Therapeutics is dedicated to creating RNA therapies that can address both rare and prevalent diseases with unmet medical needs [3] - The Axiomer technology utilizes the human cell's own ADAR machinery to make precise single nucleotide edits in RNA, potentially reversing mutations or modulating protein expression [2][3] Upcoming Events - ProQR will present at the H.C. Wainwright Genetic Medicines Virtual Conference on October 14-15, 2025, with presentations available on demand starting October 14 [5] - The company will also participate in the H.C. Wainwright Liver Disease Virtual Conference on October 21-22, 2025, and the Chardan's 9th Annual Genetic Medicines Conference on October 21, 2025 [5]

Core Insights - The company aims to significantly enhance lives through RNA therapy delivery, reflecting a strong commitment to innovation in the healthcare sector [1] Company Development - Since its IPO five years ago, the company has progressed from having no clinical programs to currently having three programs in late-stage development, indicating substantial growth and advancement in its research and development efforts [1]

Financial Data and Key Metrics Changes - The company has progressed significantly since its IPO five years ago, moving from no programs in the clinic to three programs in late-stage development [3][4] - The company plans to file three Biologics License Applications (BLAs) within a 12-month period, starting with the DMD Delzeta program for accelerated approval in the U.S. [4][24] Business Line Data and Key Metrics Changes - The Delzeta program has shown a 25% increase in dystrophin levels in patients, with a significant decrease in creatine kinase levels [11][12] - The company is focused on delivering first-in-class and best-in-class therapies across its product lines, with a strong emphasis on muscle delivery [4][10] Market Data and Key Metrics Changes - There are approximately 900 boys and young men in the U.S. with DMD exon 44, with about 50% of them treated at the same 40 centers of excellence [33] - The company anticipates that its therapies will be the first and best available options for DMD patients [34] Company Strategy and Development Direction - The company is preparing for commercialization with a foundational commercial infrastructure that can be leveraged across all three drug opportunities [9][10] - The company is exploring partnerships and M&A opportunities as it approaches the commercial phase with potential blockbuster drugs [8][9] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in executing their plans and emphasized the importance of focusing on upcoming approvals and launches [9][10] - The company is optimistic about the regulatory landscape and believes its data package will be compelling for the FDA [30][41] Other Important Information - The company has removed biopsy requirements for new participants in the Explore 44 trial, focusing on dystrophin and creatine kinase as key markers for the BLA filing [23][25] - The company is actively working on global trial enrollment for FSHD, with strong community support [46][47] Q&A Session Summary Question: Can you provide an overview of the company's platform and key programs? - The company has three programs in late-stage development and aims to improve lives through RNA therapy [3] Question: What is the commercial strategy and business development approach? - The company is building a commercial infrastructure and is open to exploring M&A opportunities [8][9] Question: Can you discuss the Delzeta program and its data? - The Delzeta program has shown a 25% increase in dystrophin levels and will present functional data ahead of schedule [11][12][16] Question: What is the market opportunity for DMD exon 44? - There are about 900 DMD exon 44 patients in the U.S., and the company plans to target this population effectively [33] Question: What are the key filing requirements for the BLA? - The primary requirements include demonstrating dystrophin levels and safety data, with a focus on creatine kinase as a critical marker [23][25] Question: Will there be a pre-BLA meeting? - Yes, a pre-BLA meeting is planned to ensure the data package meets FDA expectations [44] Question: What are the expectations for the Harbor study's primary endpoint? - The primary endpoint is video hand opening time, and the company is confident in meeting this goal [51]