The company also noted that no new safety signals were identified, reinforcing the favorable safety profile observed to date. The study, however, met the biological endpoint of micro-dystrophin protein expression at 12 weeks post-treatment, as measured by western blot, the company said. Sarepta Therapeutics Inc (NASDAQ: SRPT) announced topline results from Part 1 of the Phase 2 study, dubbed Study 102, that is evaluating its investigational gene therapy, SRP-9001, in Duchenne muscular dystrophy, showing the study did not achieve statistical significance on the primary functional endpoint of improvement in NSAA total score compared to placebo at 48 weeks post treatment. Stocks In Focus Sarepta Phase 2 Duchenne Muscular Dystrophy Gene Therapy Study Does Not Meet Primary Endpoint 7)įusion Pharmaceuticals Inc (NASDAQ: FUSN) (Biotech Stocks Hitting 52-week Lows Jan. Zai Lab Ltd – ADR (NASDAQ: ZLAB) ( announced in-licensing of Chinese rights to argenx SE's – ADR (NASDAQ: ARGX) efgartigimod) Xencor Inc (NASDAQ: XNCR) (announced a cancer drug collaboration) United Therapeutics Corporation (NASDAQ: UTHR) SAGE Therapeutics Inc (NASDAQ: SAGE) (issued a corporate strategy update) Rhythm Pharmaceuticals Inc (NASDAQ: RYTM) Praxis Precision Medicines Inc (NASDAQ: PRAX) (received rare pediatric disease and orphan drug designations for its severe pediatric epilepsy programs) Pacira Biosciences Inc (NASDAQ: PCRX)(announced above-consensus preliminary revenues for 2020) Pacific Biosciences of California Inc (NASDAQ: PACB) Oxford Immunotec Global PLC (NASDAQ: OXFD (announced a deal to be acquired by PerkinElmer, Inc. Neuronetics Inc (NASDAQ: STIM) (announced expansion of its commercial organization) Intellia Therapeutics Inc (NASDAQ: NTLA) (the genomic editing company announced 2021 milestones and strategic priorities)
IPOS 4 GENE UPDATE
(NASDAQ: BASI)Įvelo Biosciences Inc (NASDAQ: EVLO) (announced expansion of clinical program and issued an update on key 2021 milestones) (NASDAQ: AVIR)Īxonics Modulation Technologies Inc (NASDAQ: AXNX)īioanalytical Systems, Inc. (NASDAQ: ANGO) (reacted to its fiscal-year 2021 second-quarter results)Īpellis Pharmaceuticals Inc (NASDAQ: APLS)Ītea Pharmaceuticals, Inc. (Biotech Stocks Hitting 52-week Highs Jan. Beam hopes to file Investigational New Drug applications (INDs) for multiple programs this year, enabling the start of clinical studies, followed by a wave of IND filings in 2021.Here's a roundup of top developments in the biotech space over the last 24 hours. The company says it has achieved preclinical proof-of-concept for a treatment for sickle cell disease and believes it's close to achieving that milestone for other programs, including one for use with CAR-T treatments for leukemia. Where CRISPR uses genomic "scissors," Beam, in its filing with the SEC, described its approach as more analogous to a pencil that can erase and rewrite letters in the gene sequence.īeam is working on 12 different therapeutics, targeting genetic diseases in hematology, oncology, liver disease, and eye disorders. Base editing, in contrast, can precisely target a single base pair and edit the DNA "letter" without cutting the molecule. Most existing gene-editing technologies, such as CRISPR, make double-stranded cuts to DNA and rely on cellular mechanisms to repair the molecule and complete the edit. Beam Therapeutics is a preclinical-stage company that's developing a gene-editing platform using "base editing" technology, which enables changes to individual bases, the fundamental building blocks of DNA.
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