As the American Society of Gene & Cell Therapy’s 25th Annual Meeting wraps up, a host of life science companies touted their research and products. BioSpace looks at some of them.
The American Society of Gene & Cell Therapy’s 25th Annual Meeting is being held virtually and in person from May 16 to 19 in Washington DC. Here are some of the highlights.
Genascence Osteoarthritis Drug Shows Promise in Phase I Trial
Genascence Corporation shared positive safety data from its Phase I clinical trial of GNSC-001 for osteoarthritis, a degenerative joint disease, noting that the drug was well-tolerated and safe even after a 12-month follow-up on participants.
GNSC-001 is a recombinant adeno-associated vector (AAV) that carries a coding sequence for interleukin-1 receptor antagonist, which can inhibit IL-1 signaling. IL-1 is one of the primary mediators of OA and can cause cartilage destruction and inflammation. GNSC-001 is injected into the affected joint to offer long-term IL-1 inhibition.
“We are excited by these findings as they demonstrate the initial safety of GNSC-001 and provide encouraging data to pursue GNSC-001 as a novel treatment for OA patients. We look forward to advancing the clinical program for GNSC-001 so that we can deliver transformative results for patients suffering from this disabling disease,” Thomas Chalberg, Ph.D., founder and chief executive officer of Genascence, said in a statement.
Results of the Phase I trial were presented by Dr. Christopher Evans.
XyloCor Cardio Candidate Excels in Phase I
XyloCor Therapeutics announced that its proposed treatment for refractory angina had maintained positive safety, efficacy and tolerability levels six months after the Phase I dose-escalation trial began.
Initial clinical data from the Phase I part of the ongoing Phase I/II EXACT study of XC001 (encoberminogene rezmadenovec) for the said cardiovascular disease delivered positive results across all tested doses. While the first part of the trial was all about determining the drug’s safety, the initial data showed positive trends in ischemic burden and symptom reduction. These results empower the advancement to the Phase II dose selection part.
“We anticipate that the Phase II expansion portion of this study, which is testing the highest and most efficacious dose from Phase I, will complete enrollment this month. We are incredibly excited by the potential for this investigational therapy to improve the quality of life for these cardiac patients,” Thomas Povsic, M.D., Ph.D., the national principal investigator for the EXACT trial, said in a press release.
Vedere Bio II Preps for Bigger Scale Production of Ocular Gene Therapies
Vedere Bio II presented new preclinical data identifying three novel AAV capsids that would help advance its discovery efforts for ocular gene therapies.
In the ASGCT presentation titled “Novel AAV Capsids for Intravitreal Discovery: Identifying and Characterizing Novel AAV Variants in Non-Human Primates,” Vedere Bio described a directed evolution approach to overcoming the inner limiting membrane that prevents therapeutics from entering the retina. The three novel capsids can limit inflammatory response, display superior transduction efficiency to retinal cells, reduce off-target transduction and demonstrate efficient manufacturability.
“These advances not only enhance the probability of success for Vedere’s lead program but also the ability to build a pipeline and scale the production of high-quality vector preps,” Gabor Veres, Ph.D., chief scientific officer of Vedere Bio II, said.
Aside from the three AAV capsids, Vedere presented data on manufacturing AAV vectors at high titers and percent-full capsids, which are important to achieving commercial viability. Vedere’s suspension cell line X-RAP appears to consume fewer nutrients but quintuples the capsids and delivers a titer that’s ~1.8x greater than a commercially available cell line for rAAV production. The results are based on the use of a 2L bioreactor, but the company also plans to use a 50L bioreactor in the future to see if it can be applied to processes on an industrial scale.
Senti Bio Advances Cancer Pipeline
Senti Bio shared positive progress from its cancer research efforts, highlighting that many of its drug candidates are in the final stages of selection and that Investigational New Drug applications could be filed by 2023.
In a presentation at ASGCT, Senti Bio put the spotlight on its two lead programs for hepatocellular carcinoma and acute myeloid leukemia. The company also talked about its Smart Sensor gene circuits, which can sense a disease state and then activate gene expression in response. Abstracts and presentation details are available on the Senti Bio and ASGCT websites.
Locanabio’s ALS Drug Demonstrates Effectiveness in Preclinical Trial
Locanabio presented preclinical data indicating that its CORRECTx platform holds value for the possible treatment of amyotrophic lateral sclerosis (ALS). The presentation was based on both in vitro data and data from a mouse model.
Preliminary findings showed a significant reduction in toxic RNA transcripts from hexanucleotide (G4C2) repeat expansions in the chromosome 9 open reading frame 72 (C9ORF72) gene, which is understood to be closely linked with ALS. The platform comprises a CRISPR-Cas13d system with two guide RNAs that target both disease-causing sense and antisense RNA repeats. It is packaged into a single AAV9 vector for easy delivery.
“The versatility of our selective CORRECTx RNA-targeting approach provides Locanabio with several advantages as we advance development candidates from our novel class of genetic medicines to other disease targets. The data presented today supports our plans to leverage the multi-targeting and mutation specific capabilities of the CORRECTx platform to expand our pipeline and treat other serious diseases with limited or no treatment options,” James Burns, Ph.D., CEO of Locanabio, stated.
Details of the presentation titled “AAV9 mediated delivery of RNA targeting systems eliminate hexanucleotide repeat expansions in C9ORF72 ALS/FTD models” may be viewed on the Locanabio website.
CODA is Taking a Big Whack at Focal Epilepsy
This week, CODA Biotherapeutics is presenting preclinical data detailing the assessment and selection of AAV regulatory cassettes that drive optimal expression of its novel engineered ligand-gated ion channel for the treatment of focal epilepsy.
CODA’s approach to intractable focal epilepsy involves oral administration of a small molecule ligand, which can be finely tuned to control the aberrant activity of neurons and thereby suppress seizures without adverse effects. The ligand is designed to only interact with CODA’s chimeric LGIC. The selection of the AAV expression cassettes is critical as they enable the drive optimal expression of the LGIC in target cells of the hippocampus.
The findings will inform the selection process of a final candidate for this program, bringing CODA one step closer to the clinic.
