On Monday, H.C. Wainwright initiated coverage on Benitec BioPharma (NASDAQ:BNTC), a biotechnology firm focused on gene therapy, with a Buy rating and a price target of $28.00. The firm highlighted the company's proprietary Silence and Replace DNA-directed RNA interference (ddRNAi) platform as a key innovation, particularly with its lead candidate, BB-301, for the treatment of oculopharyngeal muscular dystrophy (OPMD).
OPMD is a rare and progressively degenerative genetic disorder that affects muscle function in the eyes, throat, and eventually other parts of the body. It typically emerges in adults between the ages of 40 and 60. Currently, there are no available therapies for OPMD, which can lead to difficulties in vision, swallowing, talking, and walking as the condition progresses.
BB-301 represents a novel therapy approach by co-expressing a codon-optimized Poly-A Binding Protein Nuclear-1 (PABPN1) and two small inhibitory RNAs (siRNAs) to target the mutant PABPN1 gene associated with OPMD. The therapy aims to silence the expression of the faulty gene while allowing for the expression of a functional version to replace it.
The analyst from H.C. Wainwright noted that BB-301 has already shown favorable clinical data in two patients with OPMD, with more subjects expected to provide additional data in the coming months. The firm believes that positive outcomes in a relatively small number of individuals with OPMD could support the potential approval of BB-301.
The establishment of a $28.00 price target reflects the analyst's confidence in the potential of BB-301 to become a significant treatment option for OPMD, considering the absence of current therapies and the promising early clinical results. Benitec BioPharma's focus on genetic medicine and the development of its ddRNAi platform positions the company as an emerging force in the field of gene therapy.
This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.