Benitec Biopharma Ltd. (NASDAQ: BNTC) saw its shares more than double on Tuesday after the company gave a business update on its ddRNAi technology for the development of therapeutics for the treatment of ocular diseases. Specifically Benitec noted that the output from its collaboration with 4D Molecular Therapeutics has been instrumental in the treatment’s development. Although this update does seem vague at first glance, the company appears to be planning a firework for an upcoming convention.
For some brief background: Benitec’s ddRNAi technology is a unique combination of gene silencing using RNA interference coupled with the long-term therapeutic activity of gene therapy vectors. The lead ocular candidate, BB-201, is designed to treat patients with the wet form of age-related macular degeneration (AMD) and will be featured in the ARVO-Asia presentations.
We can look forward to more information being presented about the ddRNAi technology at the Association for Research in Vision and Ophthalmology (ARVO)-Asia meeting, which will be held in Brisbane on February 5 through February 8, and the Translational Vision Summit (TVS) being held in conjunction.
Dr. David Suhy, Benitec’s chief scientific officer, commented:
Having a commercially attractive route of administration is a significant step forward in the program. One of the major limitations of most ocular gene therapy applications is the use of a highly complex surgical technique called subretinal injection for delivery into the eye. We are developing viral vectors which can efficiently transduce cells within the retina following an office-friendly, intravitreal injection. This is the same route of administration used for the standard of care treatments for AMD, including Lucentis and Eylea.
Being able to deliver drugs in therapeutically relevant concentrations is a key challenge in drug development. It has taken significant time and effort, but we believe that these outcomes demonstrate the commercial applicability of having a vector that can transduce the retina following an intravitreal injection. The AMD program is our first program in this space and we anticipate being able to build a ddRNAi franchise for other ocular indications, in particular retinal diseases, using these novel viral vectors as a key component in that platform.
Shares were last seen up over 140% at $3.88, with a consensus analyst price target of $14.00 and a 52-week trading range of $1.30 to $4.40.