NEW YORK – Quansys Biosciences and the Michael J. Fox Foundation for Parkinson's Research said this week that they are partnering to accelerate research into the disease. The deal will draw on Quansys' expertise in multiplex ELISAs and MJFF's understanding of Parkinson's to develop new immunoassays for monitoring disease progression and "target engagement for therapeutics in clinical trials." Based in Logan, Utah, Quansys develops multiplex ELISAs. Its proprietary Q-Plex technology enables researchers to access quantitative data quickly and efficiently, it said.
Taysha Gene Therapies this week said an independent data monitoring committee recommended proceeding with dosing of a second patient in the Phase I/II trial of the company's investigational Rett syndrome candidate TSHA-102, following promising initial clinical data from the first patient dosed with the gene therapy. The trial, dubbed REVEAL, is a first-in-human, open-label, randomized, dose-escalation, and dose-expansion study evaluating the safety and efficacy of TSHA-102 in adult women with Rett syndrome caused by loss-of-function mutations in the MECP2 gene. Taysha expects to dose the second patient in Q3.
Invectys on Monday said that the US Food and Drug Administration granted fast-track designation to its CAR T-cell therapy targeting human leukocyte antigen G (HLA-G). The company is investigating the safety, tolerability, pharmacokinetics, and clinical activity of IVS-3001 in a Phase I/IIa trial in patients with previously treated, locally advanced, unresectable or metastatic solid tumors that express HLA-G. Fast-track designation allows a sponsor to meet with the FDA more frequently to get advice on developing designated agents, submit data on a rolling basis, and apply for accelerated approval or priority review.
BioMarin this week said it expects Roctavian, the gene therapy for severe hemophilia A that received US Food and Drug Administration approval in June, to be available for commercial use in the US in August. In Europe, where Roctavian was granted approval in 2022, BioMarin is working with the single public insurance funds in France and Italy to secure reimbursement and access to Roctavian, which is expected later this year. The company is also finalizing access for patients in Germany. BioMarin said it expects to treat the first patients with Roctavian in the US and Europe this year.
Vertex Pharmaceuticals this week as part of a report on its second-quarter earnings said the US Food and Drug Administration has indicated plans to hold an advisory committee meeting for exagamglogene autotemcel (exa-cel), a gene-editing therapy candidate for sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT) that Vertex developed in collaboration with CRISPR Therapeutics. The FDA in June accepted the companies' biologics license applications for the two indications, including granting exa-cel priority review for SCD. The FDA is expected to issue a decision on exa-cel's SCD indication by Dec. 8 and the TDT indication by March 30, 2024.
ITM Isotope Technologies and POINT Biopharma said this week that they have expanded a global supply agreement such that ITM will supply more lutetium-177 to POINT for clinical trials and potential future commercial development of POINT's targeted radiopharmaceuticals. POINT is developing radionuclides for FAP-expressing solid tumors and for prostate-specific membrane antigen-expressing prostate cancers, among other indications. The firms did not disclose the financial details of their expanded supply agreement.
The US Food and Drug Administration this week granted orphan drug designation to ABM Therapeutics' BRAF inhibitor ABM-1310 for BRAF V600E-mutated glioblastoma patients. The FDA grants orphan designation to drugs for rare diseases, allowing sponsors to receive tax credits for clinical trials, user fee exemptions, and seven years of market exclusivity should the drug net FDA approval.
Mainz Biomed said this week that it has partnered with Marylebone Laboratory, based in London. Under the agreement, Marylebone is offering Mainz's ColoAlert at-home colorectal cancer screening test to its customers in the UK and internationally.
LUNGevity Foundation this week said it launched the Early Lung Cancer Center to accelerate development and adoption of early cancer detection and treatments for patients with lung cancer. The center will focus on screening, early-stage workflows, emerging technologies, and implementation and standardization of workflows for patients with incidental pulmonary nodules. AstraZeneca is a founding sponsor of the initiative.
In Brief This Week is a selection of news items that may be of interest to our readers but had not previously appeared in Precision Medicine Online.