The pipeline update is the latest internal changes to come to the surface at the Vertex Pharmaceuticals-partnered biotech after a round of layoffs claimed 10% of staff.
CRISPR Tx is cutting two CAR-T candidates, dubbed CTX110 and CTX130, and shifting to its “next-generation candidates,” according to a Dec. 4 release shared after market close.
CTX110 was designed to target CD19+ B-cell malignancies, while CTX130 was being examined against both solid tumors and certain hematologic malignancies. The two programs gave CRISPR Tx “proof of concept that allogeneic CAR-T cells can produce durable remissions following a standard lymphodepletion regimen,” according to the release.
But CRISPR Tx thinks next-gen candidates CTX112 and CTX131 can do better. The decision is based on early data from ongoing clinical trials that suggest the newer assets could improve the efficacy observed with the earlier candidates.
According to the biotech, the gene edits made for CTX112 and CTX131 lead to higher CAR-T cell expansion and functional persistence in patients when compared to the earlier candidates. The next-gen candidates also have better manufacturing profiles, with a higher and more consistent number of CAR-T cells created per batch.
CRISPR Tx will transition patients treated with CTX110 and CTX130 to long-term follow-up programs where necessary.
In the spotlight now is CTX112, which targets CD19 and is being studied in a phase 1/2 trial for patients with B-cell malignancies. Meanwhile, CTX131 targets CD70 and is being tested in a phase 1/2 trial for solid tumors.
The two assets include a Regnase-1 edit that is supposed to remove a “brake” on T cell function, as well as an edit to TGFBR2 designed to delete a “brake” on T cell anti-tumor activity.
CRISPR Tx also plans to launch a clinical trial for CTX112 in systemic lupus erythematosus in the first half of next year, with the potential to expand into other autoimmune indications later on. The company cited early clinical studies showing CD19-directed autologous CAR-T therapies led to durable remissions in several autoimmune conditions.
All these changes come right before the FDA is set to decide on the Vertex-partnered exa-cel, which has been submitted for approval in sickle cell, on Dec. 8.
Despite an earlier approval from U.K. regulators and a fairly positive reception from FDA advisers during an Oct. 31 meeting, CRISPR Tx was still forced to make staff cuts this November. Three sources familiar with the decision told Fierce Biotech that the layoffs were communicated on Nov. 7, and two of the sources said about 50 employees were let go—making up about 10% of the company.
PackGene Biotech is a world-leading CRO and CDMO, excelling in AAV vectors, mRNA, plasmid DNA, and lentiviral vector solutions. Our comprehensive offerings span from vector design and construction to AAV, lentivirus, and mRNA services. With a sharp focus on early-stage drug discovery, preclinical development, and cell and gene therapy trials, we deliver cost-effective, dependable, and scalable production solutions. Leveraging our groundbreaking π-alpha 293 AAV high-yield platform, we amplify AAV production by up to 10-fold, yielding up to 1e+17vg per batch to meet diverse commercial and clinical project needs. Moreover, our tailored mRNA and LNP products and services cater to every stage of drug and vaccine development, from research to GMP production, providing a seamless, end-to-end solution.
Related News
NeuExcell Therapeutics Unveils Breakthrough in Stroke Treatment at ASGCT 2024
In a groundbreaking development, NeuExcell Therapeutics has revealed a significant breakthrough in its NXL-001 product for stroke treatment. The announcement was made at the 2024 American Society of Gene and Cell Therapy (ASGCT) Annual Meeting, where the company...
Unraveling CRISPR Precision: BreakTag Illuminates Pathways to Improved Gene Editing
Introduction:The quest for precision in CRISPR-Cas9 gene editing takes a significant leap forward with the development of BreakTag, a method devised to enhance our understanding of DNA double-strand breaks (DSBs) induced by Cas9. This breakthrough, detailed in a...
Sumitomo Pharma Announces FDA Acceptance of Supplemental New Drug Application for Vibegron in Men with Overactive Bladder Symptoms Receiving Pharmacological Therapy for Benign Prostatic Hyperplasia
–Supplemental New Drug Application (sNDA) submission based on Phase 3 study of vibegron 75mg (GEMTESA) demonstrating statistically significant reductions in daily micturition and urgency episodes– –If approved, vibegron will be the first and only beta-3 agonist for...
Herpes cure with gene editing makes progress in laboratory studies
Herpes simplex virus. Credit: CDCResearchers at Fred Hutch Cancer Center have found in pre-clinical studies that an experimental gene therapy for genital and oral herpes removed 90% or more of the infection and suppressed how much virus can be released from an...
Related Services