CRISPR-Based Gene Therapy Earns Beta Thalassemia Approval CRISPR-Based Gene Therapy Earns Beta Thalassemia Approval
Exa-cel earned its second FDA approval. The one-time therapy, which has a list price of $2.2 million in the United States, should be available for patients early this year.Medscape Medical News (Source: Medscape Hematology-Oncology Headlines)
Source: Medscape Hematology-Oncology Headlines - January 17, 2024 Category: Cancer & Oncology Tags: Hematology-Oncology Source Type: news
US FDA approves Vertex/CRISPR gene therapy for an inherited blood disorder
The headquarters of US biopharmaceutical company Vertex Pharmaceuticals in Boston, Massachusets, on November 4, 2023. The U.S. health regulator has approved Vertex Pharmaceuticals and CRISPR Therapeutics' gene therapy to treat a rare blood disorder requiring regular blood transfusions, in patients…#boston #massachusets #crisprtherapeutics #vertex #oppenheimer #hartajsingh (Source: Reuters: Health)
Source: Reuters: Health - January 17, 2024 Category: Consumer Health News Source Type: news
ClearPoint Neuro gets FDA nod for SmartFrame OR Stereotactic System
ClearPoint Neuro, a global device, cell, and gene therapy-enabling company, has received 510(k) clearance from the U.S. Food and Drug Administration (FDA) for its SmartFrame OR stereotactic system for brain and spinal treatment navigation.
The system consists of two main components, SmartFrame OR and ClearPointer Optical Navigation Wand. SmartFrame OR provides stereotactic guidance for the placement and operation of instruments or devices during planning. This includes the operation of neurological procedures performed along with the use of a compatible optical stereotaxic navigation system using preoperative MR and/or CT...
Source: AuntMinnie.com Headlines - January 16, 2024 Category: Radiology Authors: AuntMinnie.com staff writers Tags: Industry News Source Type: news
BioIVT Fuels Future Cell and Gene Therapy Breakthroughs at Advanced Therapies Week
MIAMI, Jan. 16, 2024 /PRNewswire/ -- BioIVT, a global research partner and biospecimen solutions provider for drug and diagnostic development, will exhibit its latest Cell and Gene Therapy (CGT) innovations at Advanced Therapies Week, taking place at the Miami Beach Convention Center from January…#miami #genetherapy #cgt #advancedtherapies #artofcellgenetherapy #milestonefda #parijatjain #bioivt #ruo #gmp (Source: Reuters: Health)
Source: Reuters: Health - January 16, 2024 Category: Consumer Health News Source Type: news
ClearPoint Neuro Announces FDA Clearance for SmartFrame OR ™ Stereotactic System
SOLANA BEACH, Calif., Jan. 16, 2024 (GLOBE NEWSWIRE) -- ClearPoint Neuro, Inc. CLPT (the "Company"), a global device, cell, and gene therapy-enabling company offering precise navigation to the brain and spine, today announced it has received 510(k) clearance for its SmartFrame OR™ Stereotactic…#solanabeach #mrandorct #clearpointer #clearpoint #joeburnett #clearpointneuro #robrubio #fda #southamerica #cns (Source: Reuters: Health)
Source: Reuters: Health - January 16, 2024 Category: Consumer Health News Source Type: news
Atsena Therapeutics Receives Rare Pediatric Disease Designation from FDA for ATSN-101 Gene Therapy for GUCY2D-associated Leber Congenital Amaurosis (LCA1)
DURHAM, N.C., Jan. 16, 2024 (GLOBE NEWSWIRE) -- Atsena Therapeutics, a clinical-stage gene therapy company focused on bringing the life-changing power of genetic medicine to reverse or prevent blindness, today announced the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease…#atsenatherapeutics #fda #leber #gucy2d #lca1 #atsena #rarepediatricdisease #patrickritschel #mba #societymeeting (Source: Reuters: Health)
Source: Reuters: Health - January 16, 2024 Category: Consumer Health News Source Type: news
Bayer & #039;s AskBio initiates Phase II GenePHIT trial in Congestive Heart Failure (CHF)
Bayer AG and Asklepios BioPharmaceutical, Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, have announced the initiation of GenePHIT (Gene PHosphatase Inhibition Therapy), a Phase II trial of AB-1002 (also known as NAN-101) for the treatment of congestive heart failure (CHF). (Source: World Pharma News)
Source: World Pharma News - January 12, 2024 Category: Pharmaceuticals Tags: Featured Bayer Business and Industry Source Type: news
Bringing Ophthalmology Trials into Focus: Reducing Patient Burden, Improving Efficiency and Incorporating Cell and Gene Therapy, Upcoming Webinar Hosted by Xtalks
In this free webinar, learn about patient-centered approaches shaping ophthalmology trials, uniting stakeholders for better treatments and outcomes. The speakers will address advances in trial design specific to rare/genetic diseases of the eye, with a focus on cell and gene therapy, the... (Source: PRWeb: Medical Pharmaceuticals)
Source: PRWeb: Medical Pharmaceuticals - January 10, 2024 Category: Pharmaceuticals Tags: PDT Source Type: news
Regeneron CEO says the next big thing for biotech isn't AI, it's gene therapy
Regeneron CEO Leonard Schleifer elaborated on the biotech company's latest ventures with CNBC's Jim Cramer on Tuesday and emphasized the importance of genetic research across the industry. "The big thing for everybody these days is AI, that's the next big thing. But I don't believe that,"…#regeneron #leonardschleifer #jimcramer #decibeltherapeutics #glp1 (Source: Reuters: Health)
Source: Reuters: Health - January 10, 2024 Category: Consumer Health News Source Type: news
Sarepta Expects Prelim Gene Therapy Revenue To Be $131M In Q4, $200M For Fiscal Year
The Cambridge, Massachusetts-based company said preliminary Elevidys net product revenue is expected to be $131.3 million for Q4 and $200.4 million for FY23, significantly exceeding consensus. Fourth quarter and full-year 2023 net product revenue for Sarepta's RNA-based PMOs are expected to be…#cambridge #massachusetts #sarepta #wedbush #dmd #fda #elevidysdmd #srpt #boeing #max (Source: Reuters: Health)
Source: Reuters: Health - January 9, 2024 Category: Consumer Health News Source Type: news
Passage Bio shifts priorities after identifying new lead gene therapy candidate
The company is focused on developing a gene therapy treatment from frontotemporal dementia, the condition that has afflicted actor Bruce Willis. (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - January 9, 2024 Category: Biotechnology Authors: John George Source Type: news
Vertex, bluebird detail rollout plans for competing sickle-cell gene therapies
Vertex and bluebird bio, the companies behind the competing sickle-cell treatments approved in December, have provided new details in their plans to bring the treatments to patients in 2024. (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - January 8, 2024 Category: Pharmaceuticals Authors: Hannah Green Source Type: news
AskBio Phase Ib trial of AB-1005 gene therapy in patients with Parkinson & #039;s disease meets primary endpoint
Bayer AG and Asklepios BioPharmaceutical, Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, announced today the completion of the 18-month data collection in the Phase Ib clinical trial for AB-1005 (AAV2-GDNF), an investigational gene therapy for treating patients with Parkinson's disease (PD). (Source: World Pharma News)
Source: World Pharma News - January 4, 2024 Category: Pharmaceuticals Tags: Featured Bayer Business and Industry Source Type: news
Health Canada approves Pfizer's gene therapy for treatment of hemophilia
Pfizer said on Wednesday that Canada's health regulator approved its gene therapy for the treatment of a rare inherited bleeding disorder called hemophilia B ahead of a U.S. decision. (Source: CBC | Health)
Source: CBC | Health - January 3, 2024 Category: Consumer Health News Tags: News/Health Source Type: news
Novartis, Voyager sign gene therapy deal worth up to $1.3B
The companies have agreed to a collaboration and license agreement to develop gene therapies for Huntington ’s disease and spinal muscular atrophy. (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - January 2, 2024 Category: Biotechnology Authors: Hannah Green Source Type: news
