Regenxbio Good News for wet AMD On January 10, 2022, Regenxbio Inc. (RGNX) announced the initiation of ASCENT, the second of two Phase 3 pivotal trials to evaluate the efficacy and safety of subretinal delivery of RGX-314 in patients with wet age-related macular degeneration (wet AMD). RGX-314 is being investigated as a potential one-time gene therapy for . . . This content is for paid subscribers. …
Intellia Therapeutics Acquires Rewrite Therapeutics The acquisition is, indeed good news. By acquiring Rewrite Therapeutics, Intellia Therapeutics (NTLA) has inherited a versatile DNA writing platform which enables a range of innovative genome editing strategies. The acquisition is expected to further expand Intellia’s industry-leading genome editing toolbox by adding a highly complementary platform to its existing CRISPR/Cas9 and . . . This content is for paid …
Celularity Inc Celularity Inc. (CELU) announced the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for its non-genetically modified cryopreserved human placental hematopoietic stem cell-derived natural killer (NK) cell therapy, CYNK-001 created for the treatment of acute myeloid leukemia (AML). Headquartered in Florham Park, N.J., Celularity is a clinical . . . This content is for paid subscribers. Please click here to subscribe …
Ionis Pharmaceuticals and AstraZeneca in Collaboration to Treat ATTR with Eplontersen Ionis Pharmaceuticals (IONS) announced entering into a strategic collaboration agreement with AstraZeneca (
Amgen Announces Positive Results from Otezla Study for Psoriasis Amgen (AMGN) announced positive top-line results from the DISCREET trial - a Phase 3, multicenter, randomized, placebo-controlled, double-blind study to assess the efficacy of Otezla® (apremilast) in adults with moderate to severe genital psoriasis and moderate to severe plaque psoriasis. The study demonstrated that oral Otezla 30 mg twice daily achieved a . . . This content …
Regenxbio Granted Orphan Drug Designation for RGX-202 Regenxbio (RGNX) announced the U.S. FDA granted Orphan Drug Designation for RGX-202 - a potential one-time gene therapy for the treatment of Duchenne muscular dystrophy. RGX-202 is designed to deliver a novel, optimized microdystrophin transgene with a unique C-terminal domain and a . . . This content is for paid subscribers. Please click here to subscribe or here to log …
Cassava Sciences, Vertex Pharmaceuticals and Gilead Sciences All Have Good News Cassava Sciences & Alzheimer's Disease Cassava Sciences (SAVA) announced the initiation of a second Phase 3 trial of simufilam, its investigational drug for Alzheimer’s disease. This second Phase 3 study is designed to evaluate the safety and efficacy of simufilam over 78 weeks in approximately 1,000 Alzheimer’s . . . This content is for …
Gilead Sciences Collaboration with Arcus Biosciences Sends Stock Through the Roof There is no doubt that Gilead Sciences (GILD) is appreciating Arcus Biosciences (
Sangamo Therapeutics Preliminary Results Evaluating Isaralgagene Civaparvovec for Fabry Disease Sangamo Therapeutics (SGMO) announced preliminary results from the Phase 1/2 STAAR clinical study evaluating isaralgagene civaparvovec, or ST-920 - a wholly owned gene therapy product candidate, for Fabry disease. As of the September 17, 2021 cutoff date, results from the four patients treated in the first two dose cohorts (0.5e13 vg . . . This content is for …
Merck Stock Soared Great expectations for Merck (MRK) future growth and the future of novel treatments for patients with severe devastating and life-threatening diseases. Keytruda - Merck's cancer immunotherapy drug, has reached a milestone and molnupiravir - its COVID-19 pill, sales revenues are expected to generate $5 billion to $7 billion through 2022, subject to emergency use authorization, at the end of November 2021. The …
Intellia Therapeutics Granted Orphan Drug Designation for NTLA-2001 Intellia Therapeutics (NTLA) announced that the U.S. FDA has granted orphan drug designation to NTLA-2001 for the treatment of transthyretin (ATTR) amyloidosis. This investigational therapy is the first CRISPR therapy to be administered systemically to edit a disease-causing gene inside the human body. NTLA-2001 has the potential to be the first single-dose treatment for ATTR amyloidosis as it …
Rocket Pharmaceuticals Inc Announces Successful Results at the ESGCT Gene therapy and gene editing plus other gene and genome-based therapeutics are the only treatments expected to lead to miraculous cures. While genetic manipulation for the treatment of severe life-threatening diseases is still moving towards perfection, gene therapy and gene editing are accomplishing miraculous long-term effective treatments, saving thousands of lives where there are no currently …
Intellia Therapeutics and SparingVision Collaboration Intellia Therapeutics (NTLA) and SparingVision - a genomic medicine company developing vision saving treatments for ocular diseases, announced a strategic collaboration to develop novel genomic medicines utilizing CRISPR/Cas9 technology for the treatment of ocular diseases. Intellia Therapeutics is to grant SparingVision exclusive rights to Intellia . . . This content is for paid subscribers. Please click here to subscribe or here to …
Regenxbio Announced Data from Phase 2 ALTITUDE Trial of RGX-314 for DR Regenxbio (RGNX) announced initial data from the ongoing Phase 2 ALTITUDE™ trial of RGX-314 for the treatment of diabetic retinopathy (DR) without center-involved diabetic macular edema (CI-DME) using in-office suprachoroidal delivery. Regenxbio is a clinical-stage biotechnology company seeking to improve lives . . . This content is for paid subscribers. Please click here to …
Denali Therapeutics News Denali Therapeutics (DNLI) - a biopharmaceutical company developing a broad portfolio of product candidates engineered to cross the blood-brain barrier (BBB) for neurodegenerative diseases. Denali pursues new treatments by rigorously assessing genetically validated targets, engineering delivery across the BBB and guiding development through biomarkers that demonstrate target and pathway engagement. Denali is based in South San . . . This content is for …
Two Clinical-Stage Firms Rallied Yesterday On a day when the Stock Market was crushed, taking down with it shareholders’ money for no reason other than the self-serving untouchable agendas, two small biotechnology companies staged rallies. Atea Pharmaceuticals and Xenon Pharmaceuticals deserved the boosting of their stocks. Atea Pharmaceuticals Atea Pharmaceuticals (AVIR) - a clinical-stage biopharmaceutical company, reported for the first time . . . …
Merck and Ridgeback Biotherapeutics COVID-19 News Merck (MRK) and Ridgeback Biotherapeutics today announced that molnupiravir (MK-4482, EIDD-2801) - an investigational oral antiviral medicine, has significantly reduced the risk of hospitalization or death at a planned interim analysis of the Phase 3 MOVe-OUT trial in at risk, non-hospitalized . . . This content is for paid subscribers. Please click here to subscribe or here to log in.
Valneva SE and Pfizer Inc Announced More Positive Trial Results of VLA15 for Lyme Disease What is Lyme Disease? What is the vaccine? What is the importance of the vaccine and the company that created it? Lyme disease is an infection caused by Borrelia burgdorferi bacteria transmitted to humans by infected Ixodes ticks. Lyme disease is considered the most common vector- borne illness in the Northern Hemisphere. The U.S. …
Regeneron Welcomes the World Health Organization's Recommendation for Use of REGEN- COVTM to Treat COVID-19 The World Health Organization (WHO) updated its Therapeutics and COVID-19: living guideline to include the casirivimab and imdevimab monoclonal antibody cocktail, known as REGEN-COVTM in the U.S. and Ronapreve® in other countries. The guideline conditionally recommends REGEN-COV to treat high-risk non-hospitalized patients with non-severe COVID-19 and seronegative (no measurable . …
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