Feature|Videos|July 18, 2024
Navigating Challenges and Embracing Opportunities to Optimize mRNA Therapies
The discussion covers the challenges of optimizing mRNA therapies, including the diverse behaviors of mRNA in biological systems, as well as the opportunities presented by emerging mRNA technologies like circular and self-amplifying RNAs, and the role of regulators in ensuring quality control of these novel products.
Advertisement
Episodes in this series
The expert panel discussed the diversity of mRNA structures and behaviors, as well as the challenges and opportunities in optimizing mRNA design and synthesis. New directions in mRNA structure and chemistry, such as circular RNAs and self-amplifying RNAs, were also discussed, as well as the increasing complexity of mRNA therapeutics from a regulatory perspective.
Discussion Topics
- 00:07 - Challenges and opportunities in optimizing mRNA therapies
- 01:44 - Emerging mRNA technologies
- 04:20 - Role of regulators
Advertisement
Related to this article
A real-world study found evolocumab cut fatal or nonfatal heart attack risk 20% vs ezetimibe in adults with ASCVD and prior statin use.
FDA grants accelerated approval to Johnson & Johnson’s subcutaneous EGFR/MET bispecific Rybrevant Faspro for previously treated head and neck squamous cell carcinoma, T-Therapeutics raises a $133 million Series B to take TCR-CD3 bispecifics into clinical trials, and Alethio Therapeutics raises a $90 million Series A to advance its first-in-class G6B antibody for essential thrombocythemia.
Johnson & Johnson's amivantamab (Rybrevant Faspro) won accelerated FDA approval for pretreated recurrent head and neck cancer.
Oxford spinout Alethio Therapeutics closed a $90 million Series A to take ATX-011, a monoclonal antibody against the G6B receptor, into Phase 1/2a studies in essential thrombocythemia. It is also developing a G6B-targeted ADC for myelofibrosis.
T-Therapeutics closed an oversubscribed $133 million Series B backed by Eli Lilly, JJDC, and others. The funding will take its first T-cell receptor bispecifics in cancer and autoimmune disease into clinical trials.
China’s NMPA grants priority review to Akeso’s PD-1/VEGF bispecific ivonescimab with chemotherapy for first-line biliary tract cancer, VeonGen Therapeutics aligns with FDA on a phase 3 design for its dual-AAV Stargardt gene therapy VG801, and Caldera Therapeutics’ IL-23p19×TL1A bispecific CLD-423 shows a half-life of more than 40 days in a phase 1a trial.
FDA’s draft framework would let sponsors add patient-specific genome editing variants to an approved BLA using in vitro data. Here is what the guidance proposes, what evidence it asks for, and what remains unresolved.
Healthy-volunteer data show sustained TL1A suppression and a profile supporting infrequent subcutaneous dosing. Caldera also licensed a second bispecific, targeting IL-17 and TL1A, from Qyuns Therapeutics.
Advertisement
Advertisement
Trending on BioPharm International
1
Spyre’s Anti-TL1A Antibody SPY072 Misses in Psoriatic Arthritis and Axial Spondyloarthritis, Shifts Focus to Hidradenitis Suppurativa
2
Boehringer Ingelheim Licenses Six Preclinical ADC and T-Cell Engager Programs From SBP Group in Deal Worth Up to $7 Billion
3
The BioPharm Brief: Ivonescimab Priority Review in China, Stargardt Gene Therapy Moves Toward Phase 3, and Longer Dosing Intervals for an IBD Bispecific
4
Caldera’s IL-23p19×TL1A Bispecific CLD-423 Shows 40-Day Half-Life in Phase 1a, Supporting Dosing Every 8 to 12 Weeks
5