On May 13, 2024 Aldevron, a leading global manufacturer of DNA, RNA, and proteins used in cell and gene therapies and vaccine development, reported it has entered a strategic partnership with Acuitas Therapeutics, Inc., a private biotechnology company specializing in the development of delivery systems for nucleic acid therapeutics based on lipid nanoparticles (LNPs) (Press release, Aldevron, MAY 13, 2024, View Source [SID1234643176]). This partnership will allow Aldevron to expand its capabilities in mRNA LNP encapsulation by incorporating Acuitas’ proprietary LNP encapsulation platform, increasing Aldevron’s services and capabilities as an mRNA sequence-to-vial custom manufacturer.

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In May of 2023, Aldevron announced the expansion of its mRNA production capabilities to include Cytiva’s mRNA LNP encapsulation and aseptic fill-finish platform. This new strategic partnership with Acuitas Therapeutics, working with its encapsulation platform, equips Aldevron with a diverse range of mRNA LNP technologies, significantly enhancing its mRNA end-to-end drug product service capabilities. The partnership will enable Aldevron to support Acuitas’ LNP formulations and licensees.

"This collaboration with Acuitas is an exciting addition to our existing mRNA sequence-to-vial services and will enable Acuitas partners to leverage Aldevron’s extensive mRNA manufacturing experience and comprehensive end-to-end capabilities," said Mark Wetzel, Vice President mRNA CDMO at Aldevron. "We look forward to our continued partnership that is poised to enhance the landscape of mRNA-based therapies, bringing faster timelines and hope, to patients worldwide."

"We have long known of Aldevron’s reputation and broad experience in the manufacturing of nucleic acids and their commitment to quality," said Chris Barbosa, Vice President of Technology Development at Acuitas Therapeutics, Inc. "We are pleased to work with Aldevron to provide Acuitas partners with an integrated path to clinical drug products using Acuitas’ lipid nanoparticle manufacturing processes." He added, "We look forward to Aldevron’s contribution to working with us to support our global partners in bringing new mRNA-based therapies to patients as quickly and efficiently as possible."

"The Acuitas technology, combined with Aldevron’s extensive experience providing cutting-edge solutions for advancing therapies and rigorous quality processes, provides an exceptional advantage," continued Wetzel. "With our two decades of experience in mRNA technologies, Aldevron has developed a seamless workflow from mRNA sequence-to-vial, and this partnership provides clients LNP platform flexibility for the appropriate development stage for their program."

On May 13, 2024 Checkpoint Therapeutics, Inc. ("Checkpoint") (Nasdaq: CKPT), a clinical-stage immunotherapy and targeted oncology company, reported that James Oliviero, President and Chief Executive Officer, will participate in a fireside chat at the H.C. Wainwright 2nd Annual BioConnect Investor Conference at NASDAQ, taking place on Monday, May 20, 2024, at 12:30 p.m. ET (Press release, Checkpoint Therapeutics, MAY 13, 2024, View Source [SID1234643138]). Checkpoint will also attend in-person one-on-one meetings during the conference.

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A webcast of the fireside chat will be available on the News & Events page, located within the Investors section of Checkpoint’s website, View Source, for approximately 30 days after the meeting.

On May 13, 2024 Cartesian Therapeutics, Inc. (NASDAQ:RNAC) (the "Company), a clinical-stage biotechnology company pioneering mRNA cell therapy for autoimmune diseases, reported that management will participate in a fireside chat at the H.C. Wainwright 2nd Annual BioConnect Investor Conference on Monday, May 20, 2024, at 3:00 p.m. ET (Press release, Cartesian Therapeutics, MAY 13, 2024, View Source [SID1234643155]).

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A live webcast of the fireside chat is expected to be accessible in the Events section of the Company’s website at www.cartesiantherapeutics.com, where an archived replay of the event will be accessible for a limited time.

On May 13, 2024 Pheast Therapeutics ("Pheast"), a biotech developing novel macrophage checkpoint therapies to defy cancer, reported the first presentation of preclinical data for PHST001, an anti-CD24 antibody drug candidate that is designed to block a key macrophage "don’t eat me" signal on cancer cells (Press release, Pheast Therapeutics, MAY 13, 2024, View Source [SID1234643177]). The data to be presented at the 20th Annual PEGS Boston Summit demonstrate that PHST001 can powerfully induce an anti-cancer immune response and drive therapeutic efficacy in in vivo models.

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"Pheast’s lead drug candidate, PHST001, is an exceptionally well-engineered, well-expressed antibody against an exciting new target, CD24," said Roy Maute, Ph.D., Cofounder and CEO, Pheast Therapeutics. "Our preclinical studies show potent efficacy in challenging mouse model systems, which supports the possibility of clinical efficacy in cancers where other immunotherapies have failed and the unmet need remains high. The Pheast team is making rapid progress to support filing an investigational new drug application with the goal to enter the clinic in patients in the first half of next year."

CD24 is a newly discovered immune checkpoint that is highly expressed by many human cancers, including ovarian and triple negative breast cancer (TNBC). High expression of CD24 is a negative prognostic factor in multiple cancer indications. CD24 interacts with the macrophage receptor Siglec-10, and shields cancer cells from attack by macrophages. Pheast has engineered PHST001 to bind recombinant CD24 and cell surface CD24 with high affinity and specificity and to block Siglec-10 binding.

Pheast scientists have demonstrated that blocking CD24 with PHST001 induces macrophage phagocytosis of multiple cancer subtypes in vitro, and have shown potent efficacy for PHST001 in vivo. In some preclinical models, PHST001 monotherapy is sufficient to shrink and fully eradicate tumors in all treated mice.

"These data demonstrate Pheast’s robust in-house antibody discovery and protein engineering capabilities, allowing rapid discovery and development of therapeutic monoclonal antibodies," said John Burg, Ph.D., Senior Director, Protein Sciences. "Our innovative platform for discovery of tumor-specific immune checkpoints for precision immuno-oncology treatments will support exploring additional targets and developing new pipeline programs."

Dr. Burg will present these data on Monday, May 13, 2024, at 1:15 pm ET in a talk titled, "Engineering a Therapeutic Monoclonal Antibody Targeting CD24, a Macrophage Checkpoint."

On May 13, 2024 Circio Holding ASA (OSE: CRNA), a biotechnology company developing circular RNA-based gene therapy, reported that it has presented two posters that demonstrate in vivo proof-of-concept for its powerful and differentiated circVec platform approach to gene therapy (Press release, Circio, MAY 13, 2024, View Source [SID1234643139]). The two posters were presented at the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 2024 annual meeting 7-11 May in Baltimore, USA

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"Circio has generated results demonstrating that the circVec 2.1 design performs very well in vitro. We have now confirmed this in vivo with statistically significant higher expression level and durability for circVec 2.1 DNA vectors compared to standard linear mRNA-based expression. These results provide an important technical proof-of-concept for Circio´s technology platform in an animal model. We now have confirmation for our expectation that this could translate into improved gene therapies for patients in the future," said Dr. Thomas B Hansen, CTO at Circio. "In recent experiments, Circio has observed up to four months circVec durability in vivo. This substantially outperforms mRNA vector expression. Following these results, we can rapidly advance to design and test circVec in several AAV and DNA-based vectors. This will validate these very promising data in therapeutically relevant formats."

At ASGCT (Free ASGCT Whitepaper), Circio also presented the dual-function ‘remove-&-replace’ concept for Alpha-1-antitrypsin deficiency (AATD). This genetic disease causes severe symptoms in the lung and liver. There are currently no satisfactory therapeutic options available for this indication and AATD still represents a major unmet medical need. There are over 200,000 AATD patients affected in the USA and EU alone. With the technologically differentiated circVec remove-&-replace format, Circio has developed a unique gene therapy concept that can deal with both the lung and liver-associated symptoms in one single therapeutic.

"AATD is a challenging genetic disease to treat. This is in part due to the two distinct pathologies in the liver and lung," said Dr. Victor Levitsky, CSO at Circio. "We have now established and technically validated circVec constructs that can both replenish functional wild-type AAT and specifically remove more than 90% of the mutated protein. This is challenging to achieve because the functional and mutant forms are very similar. By using circular RNA-based AAT expression, Circio is uniquely able to separate the two species for mutant-specific knockdown, thereby solving two problems with one single product."

Optimization and In Vivo Performance of circVec, a Vector-Based Circular RNA Expression Platform; O´Leary et al. ASGCT (Free ASGCT Whitepaper) 2024

Expressing AAT from circular RNA-encoding vectors as a promising gene therapy approach for Alpha 1-antitrypsin deficiency; O´Leary et al. ASGCT (Free ASGCT Whitepaper) 2024