Category: Impacting News

Celularity Inc NK Cell Therapy CYNK-001 Receives US FDA Fast Track Designation for AML Treatment

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 …

Amgen: Positive Results from Otezla® Phase 3 DISCREET Study in Moderate to Severe Genital Psoriasis

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 …

The FDA Granted Orphan Drug Designation to Regenxbio Investigational Gene Therapy Drug RGX-202 for Duchenne Muscular Dystrophy

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 …

Let’s Talk About 3 Firms with Important News: Cassava Sciences, Vertex Pharmaceuticals and Gilead Sciences

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 …

Sangamo Therapeutics: Preliminary Data Shows Tolerability and Sustained Elevated α-Gal A Enzyme Activity in Patients with Fabry Disease

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 …

Why Merck’s Stock Price Soared. See Also: Pulmotect Inhaled PUL-042 for Respiratory Symptoms of COVID-19 and Fujirebio Agreement with ADDF

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 Receives U.S. FDA Orphan Drug Designation for NTLA-2001 See Also: Important News from Ultragenyx Pharmaceutical

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 Data Updates at the ESGCT Conference for RP-L201 for the Treatment of LAD-I

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 Strategic Collaboration to Develop Novel Ocular Therapies. See Also: Crispr Therapeutics’ News is Good, Not Bad

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 Good News About RGX-314 for Diabetic Retinopathy

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: Positive Clinical Results and Regulatory Progress for Development Programs in ALS. See Also: NTLA Tumbling Today

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 …

Atea Pharmaceuticals and Xenon Pharmaceuticals: Two Clinical-Stage Firms That Rallied Instead of Crashing Yesterday

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 Announced Successful Phase 3 Trial Results of Molnupiravir for Mild or Moderate COVID-19

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 Announce Further Positive Results in Preventing Lyme Disease 

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. …

The World Health Organization Recommends Regeneron Pharmaceuticals Antibody Cocktail for COVID-19

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 . …

Gilead Sciences: New Data Demonstrate Trodelvy Survival Benefit in Metastatic TNBC. See Also: Galapagos NV News

Gilead Sciences New Data from Trodelvy® Study Gilead Sciences (GILD) has recently announced new data from the Phase 3 ASCENT study evaluating Trodelvy® in patients with relapsed or refractory metastatic triple-negative breast cancer (TNBC) who received two or more prior systemic therapies, at least one of them for metastatic disease. Trodelvy has shown to improve progression-free survival (PFS . . . This content is for paid …

BioMarin Pharmaceutical Inc Product VOXZOGO® is the First to Prevent Dwarfism in Europe

The most important news in biological sciences is the high degree of success the biological revolution can reach. We can feel the great value every time an untreatable, devastating condition is successfully treated thanks to discoveries made possible through the information brought in by the unparalleled revolution that allows for a better understanding of the living body mechanisms of actions in health and in diseases. …