Category: Impacting News

Vertex and TreeFrog Therapeutics Announced Licensing Agreement and Collaboration to Optimize Production of Vertex Cell Therapies for Type 1 Diabetes

Vertex Pharmaceuticals and TreeFrog Therapeutics in the NEWS Vertex Pharmaceuticals  (VRTX) and TreeFrog Therapeutics announced that Vertex has obtained an exclusive license to TreeFrog’s proprietary cell manufacturing technology, C-StemTM, to optimize production of Vertex’s cell therapies for Type 1 diabetes (T1D). TreeFrog and Vertex will collaborate to scale up TreeFrog’s process to produce and amplify cells for . . . This content is for paid …

Voyager Therapeutics: Demonstrating Productivity in Collaboration with Neurocrine to Advance Gene Therapies for Neurological Diseases

Voyager Therapeutics  Voyager Therapeutics (VYGR) is a biotech company dedicated to leveraging the power of human genetics to modify the course of – and ultimately cure – neurological diseases. The firm’s pipeline includes programs for Alzheimer’s disease, amyotrophic lateral sclerosis (ALS), Parkinson’s disease, and multiple other diseases of the central nervous system. Many of Voyager programs are derived from its TRACERTM . . . This …

Syndax Pharmaceuticals: Positive Pediatric Data from Pivotal Trial of Revumenib in Relapsed/Refractory KMT2Ar Acute Leukemia at ASPHO Plenary Session

Syndax Pharmaceuticals in the NEWS Yesterday, Syndax Pharmaceuticals (NASDAQ: SNDX) announced the presentation of positive data from the pivotal AUGMENT-101 trial in pediatric patients with relapsed/refractory (R/R) KMT2A-rearranged (KMT2Ar) acute myeloid leukemia (AML) and acute lymphoid leukemia (ALL) treated with revumenib, a first-in-class menin inhibitor. The pediatric data was featured in . . . This content is for paid subscribers. Please click here to subscribe or …

Verrica Pharmaceuticals Announced the FDA Granted YCANTHTM New Chemical Entity Status and Orange Book Listing

We are currently working to bring into the upcoming ProhostBiotech Letter some small clinical and commercial innovating firms that are challenging the Top-Tier firms that are currently generating billions of dollars in selling revenues.  These firms are developers of products for treating  type 2 diabetes, obesity and NASH diseases, in addition to clinical stage firms that are challenging the current developers of gene editing diseases …

Madrigal Pharmaceuticals Announces FDA Approval of Rezdiffra for the Patients with NASH That Have Moderate to Advanced Liver Fibrosis

Madrigal Pharmaceuticals in the NEWS Yesterday, March 14, 2024 Madrigal Pharmaceuticals (MDGL) announced that the U.S. FDA has granted accelerated approval for the firm’s product Rezdiffra (resmetirom) in conjunction with diet and exercise for the treatment of adults with noncirrhotic NASH with moderate to advanced liver fibrosis (consistent with stages F2 to F3 fibrosis). The Approval for this . . . This content is for …

Arcturus Therapeutics Reports Fourth Quarter and Full Year 2023 Financial Results, Provides Corporate Update and Hosts a Conference Call

Arcturus Therapeutics Fourth Quarter and Full Year 2023 Earnings Conference Call Before listening to the Conference call, which is at 4.30 PM, we remind about Arcturus Therapeutics Holding's history, which we believe proves the firm creates novel, safe and far reaching treatments and vaccines.    Conference Call: Today, Thursday, March 7, 2024 @ 4:30 p.m. ET Domestic: 1-877-407-0784  International:1-201-689-8560 Conference ID: 13744044  Webcast Link Arcturus …

CRISPR Therapeutics Financial Results for Fourth Quarter and Full Year Ended December 31, 2023 Plus All You Need To Know About this Firm’s Capabilities   

CRISPR Therapeutics Since its inception over a decade ago, CRISPR Therapeutics (CRSP) has transformed from a research-stage company advancing programs in the field of gene editing, to a company with a diverse portfolio of product candidates across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine, cardiovascular and rare diseases. The Nobel Prize-winning CRISPR science has revolutionized biomedical research and represents . . …

CRISPR Therapeutics to Present at the Citi 2024 Virtual Oncology Leadership Summit. See Also: CRISPR Therapeutics Advanced Steps

CRISPR Therapeutics Announcement Today, Feb. 15, 2024, CRISPR Therapeutics announced that members of its senior management team will present at the Citi 2024 Virtual Oncology Leadership Summit on Wednesday, Feb 21, 2024, at 1:00 p.m. ET. A live webcast of the fireside chat will be available on Events & Presentations page in the Investors section of the Company's website. A replay of the webcast will …

Regenxbio Product RGX-121 for the Treatment of MPS II Achieves Primary Endpoint

Regenxbio Product RGX-121 Achieves Primary Endpoint From Regenxbio (RGNX) we learned that results from the Phase I/II/III CAMPSIITE® trial of the firm’s product RGX-121 for the treatment of 5 years old patients diagnosed with Mucopolysaccharidosis Type II (MPS II), also known as Hunter syndrome, demonstrated that the pivotal phase of the trial met its primary . . . This content is for paid subscribers. Please …

Apellis Announces Negative CHMP Opinion on Pegcetacoplan for GA in the EU and Its Plans to Seek Re-Examination of Application

Apellis Pharmaceuticals Negative CHMP Opinion Apellis Pharmaceuticals (APLS) announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has adopted a negative opinion on the marketing authorization application (MAA) of intravitreal pegcetacoplan for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD). This opinion was expected based . . . This content is for paid subscribers. …

Eli Lilly Gene Therapy Restores Patient Hearing

Akous Inc to Present at the Association for Research in Otolaryngology Akouos, Inc. - a wholly owned subsidiary of Eli Lilly and Company (LLY) announced positive initial clinical results from the Phase 1/2 AK-OTOF-101 study, which restored the hearing within 30 days of AK-OTOF administration in the first participant, with more than a decade history of deep . . . This content is for paid …

Yes, Compugen’s Stock is Soaring, But Why?

Compugen's Soaring Stock Many recent events  led to Compugen's (CGEN) soaring stock after a huge decline in its price. In spite of the tremendous stock decline, Prohost never doubted the fact that Compugen has solid scientific fundamentals and great scientists. The firm has done a great  job utilizing its broadly applicable predictive computational discovery capabilities to identify new drug targets and biological . . . …

Cytokinetics Announces Positive Results from a Pivotal Phase 3 Clinical Trial of Aficamten

Cytokinetics We recently became enthusiastic about Cytokinetics’ (CYTK) current clinical trial results. A few days ago, Cytokinetics announced positive results from SEQUOIA-HCM (Safety, Efficacy, and Quantitative Understanding of Obstruction Impact of Aficamten in HCM), the pivotal Phase 3 clinical trial of aficamten in patients with symptomatic obstructive hypertrophic cardiomyopathy (HCM). Why the Great Enthusiasm? The following has been behind our enthusiasm . . . This …

Sarepta Therapeutics Submits Efficacy Supplement to Expand the ELEVIDYS Label

Sarepta Therapeutics in the NEWS On 12, 22, 2023, Sarepta Therapeutics (SRPT) - a precision genetic medicine for rare diseases, announced submission of an efficacy supplement to the Biologics License Application (BLA) for ELEVIDYS (delandistrogene moxeparvovec-rokl) to expand its labeled indication as follows: “ELEVIDYS is indicated for] the treatment of Duchenne muscular dystrophy (DMD) patients with a confirmed . . . This content is for paid …
Why Arcturus Therapeutics Holdings’ Pipeline Products are Promising

Why Arcturus Therapeutics Holdings’ Pipeline Products are Promising

The information is the first of a few biotech Companies that we believe are promising in treating severe devastating  untreatable sicknesses.  Arcturus Therapeutics Holdings Based in San Diego, California, Arcturus Therapeutics Holdings (ARCT) was founded in 2013. The Company is a late-stage clinical developing novel mRNA medicines and vaccines which already  demonstrated promising results in early trials. The Company is creating . . . This content …

Anavex Life Sciences Product Blarcamesine for Alzheimer’s Disease Receives Promising News from the EMA

Anavex Life Sciences Promising News From Anavex Life Sciences  (AVXL) we learned that The Committee for Medicinal Products for Human Use (CHMP) within the European Medicines agency (EMA) agreed that oral blarcamesine for Alzheimer’s disease is eligible for submission of application for a Union Marketing Authorization in the EU under the European Medicines Agency’s centralized procedure . . . This content is for paid subscribers. Please click here …