Rocket Pharmaceuticals Inc. has received IND clearance from the FDA for RP-A601, an AAV.rh74-based gene therapy candidate for the treatment of arrhythmogenic cardiomyopathy due to plakophilin 2 pathogenic variants (PKP2-ACM). RP-A601 offers the potential for a one-time, curative alternative to medical therapy, implantable cardioverter defibrillators and ablations.
Suzhou Genassist Therapeutic Co. Ltd. has announced its pre-IND application of its first base-editing product, GEN-6050, and acceptance by the FDA. GEN-6050 is an in vivo base-editing drug that targets exon 50 skipping in the Duchenne muscular dystrophy (DMD) gene.
Tonix Pharmaceuticals Holding Corp. has received FDA clearance of its IND application to support a phase I trial with TNX-1500, an anti-CD40L monoclonal antibody. The first indication Tonix is seeking for TNX-1500 is the prevention of organ rejection in patients receiving a kidney transplant. Enrollment in the phase I study is expected to open in the third quarter of this year.
Onquality Pharmaceuticals LLC has received FDA clearance of its IND application for OQL-036, a potential prophylactic treatment for capecitabine-induced hand-foot syndrome (HFS).
Atsena Therapeutics Inc.’s IND application for ATSN-201 has been cleared by the FDA enabling the company to initiate a phase I/II trial in patients with X-linked retinoschisis (XLRS) caused by pathogenic or likely pathogenic mutations in RS1.
Rnaimmune Inc., a nonwholly owned subsidiary of Sirnaomics Ltd., has received clearance from the FDA for its IND application to conduct a phase I trial for RV-1730, a SARS-CoV-2 vaccine booster candidate.
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