Release Date: May 08, 2024
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
Positive Points
- Completed enrollment in the adult cohort of the Ruby clinical trial and initiated enrollment in the adolescent cohort, showing strong interest and momentum.
- On track to present substantial clinical data sets for both sickle cell disease and beta-thalassemia in mid-2024, with further updates by year-end.
- Promoted Greg Whitehead to Chief Technology and Quality Officer, enhancing leadership in technical development and operations.
- Extended collaboration with Bristol-Myers Squibb, demonstrating continued partnership success and potential for future growth.
- Strong financial position with cash reserves expected to fund operations into 2026, highlighting effective financial management and strategic planning.
Negative Points
- Facing intense competition in the gene editing space, particularly from companies with approved therapies for sickle cell disease.
- Dependent on the outcomes of ongoing patent litigation which could impact the company's intellectual property rights and commercial potential.
- High costs associated with advancing clinical trials and scaling up manufacturing capabilities.
- Regulatory challenges and uncertainties that could affect the timing and success of product approvals.
- Potential market saturation and adoption challenges for new gene editing therapies, given existing treatments and emerging alternatives.
Q & A Highlights
Q: What will be the bar for success in your in vivo pipeline proof of concept expected by the end of this year?
A: Linda Burkly, EVP, Chief Scientific Officer, mentioned they are looking for high efficiency delivery and editing for their target of interest in vivo. This will provide confidence in their ability to target the target of interest, with more details to be shared later.
Q: Can you provide some color on dosing of the pivotal cohort in the red cell program?
A: Gilmore O’Neill, CEO, President, responded that they have completed adult enrollment and scheduled many patients for dosing, with further updates to be provided later. Baisong Mei, EVP, Chief Medical Officer, added that they have alignment with the FDA on the study being a Phase 1/2/3 trial for BLA filing.
Q: Regarding the red cell program, what should we look for from treated patients in terms of safety and efficacy moving forward?
A: Baisong Mei explained that they expect to have data from at least 18 patients in the Ruby study, which will provide meaningful insights into the direction, increase of total hemoglobin, and durability of the study.
Q: What are the key factors for potential differentiated durability with 100% control in your genomic therapy for sickle cell?
A: Baisong Mei highlighted the importance of normalization of total hemoglobin and correction of anemia as potential points of differentiation for their product, Renovia.
Q: How are you thinking about future IP monetization opportunities following the agreement with Vertex?
A: Erick Lucera, EVP, CFO, stated that they view future potential royalty monetization and licensing activities as an important source of non-dilutive capital, emphasizing the foundational nature of their IP patents.
Q: What's your latest thinking around the timing of holding discussions with regulators on sickle cell, given the data you're going to present mid-year?
A: Baisong Mei mentioned ongoing engagement with the FDA, noting that they have alignment that the Ruby study is a Phase 3 study to support the BLA, with continuous discussions about the regulatory path forward.
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
