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FAQ: Antisense Oligonucleotides and Their Impact on Drug Development Economics
TL;DR
Oncotelic Therapeutics' OT-101 offers a competitive edge as the only TGF-β2-specific antisense in Phase 3 trials, targeting resistant cancers like pancreatic cancer.
Antisense oligonucleotides work by using rationally designed synthetic DNA or RNA strands to silence disease-causing genes, improving drug approval rates from 5-10% to recent FDA successes.
ASO therapies accelerate treatment development for resistant cancers, potentially saving lives by delivering new options years faster than traditional small molecule drugs.
Six new antisense drugs gained FDA approval in 2023-2024, showing how rational design is transforming drug development economics and regulatory momentum.
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Antisense oligonucleotides are short synthetic strands of DNA or RNA that work by silencing disease-causing genes through rational design, which is shifting the odds in drug development compared to traditional approaches.
While small molecules achieve only 5-10% approval rates over 15-20 years (with oncology drugs at just 3%), ASOs are experiencing accelerating regulatory momentum with six new FDA approvals in 2023-2024 alone, bringing total approvals above 20.
Oncotelic Therapeutics Inc. (OTCQB: OTLC) aims to be at the forefront with OT-101 (Trabedersen), which is the only TGF-β2-specific antisense therapy currently in Phase 3 trials, targeting pancreatic cancer and other resistant malignancies.
This is significant because traditional drug development has created a bottleneck where about 90% of candidates fail, leaving patients waiting years for new treatments that rarely arrive, while ASOs offer improved odds through rational design.
Evidence includes six new FDA approvals in 2023-2024, more than 50 candidates in active trials, and total antisense drug approvals now exceeding 20, indicating a long-anticipated breakout for the technology.
OT-101 (Trabedersen) targets pancreatic cancer and other resistant malignancies as the only TGF-β2-specific antisense therapy currently in Phase 3 trials.
The latest news and updates relating to OTLC are available in the company's newsroom at ibn.fm/OTLC.
This information was published by BioMedWire (BMW), a specialized communications platform focused on biotechnology, biomedical sciences, and life sciences sectors, which is part of the Dynamic Brand Portfolio at IBN.
Curated from InvestorBrandNetwork (IBN)
