How Jennifer Doudna ’s Life Has Changed Since Discovering CRISPR 10 Years Ago
Jennifer Doudna was staring at a computer screen filled with a string of As, Cs, Ts, and Gs—the letters that make up human DNA—and witnessing a debilitating genetic disease being cured right before her eyes. Just a year earlier, in 2012, she and microbiologist Emmanuelle Charpentier had published a landmark paper describing CRISPR-Cas9, a molecular version of autocorrect for DNA, and she was seeing one the first demonstrations of CRISPR’s power to cure a human disease. She was in the lab of Dr. Kiran Musunuru, a Harvard researcher who was eager to show her the results from an experiment he had just finish...
Source: TIME: Health - July 1, 2022 Category: Consumer Health News Authors: Alice Park Tags: Uncategorized feature Genetics healthscienceclimate Source Type: news

FDA OKs Vutrisiran for hATTR Amyloidosis With Polyneuropathy FDA OKs Vutrisiran for hATTR Amyloidosis With Polyneuropathy
Vutrisiran, an RNAi therapeutic given by subcutaneous injection once every 3 months, significantly improved signs and symptoms of hATTR polyneuropathy in a phase 3 study.FDA Approvals (Source: Medscape Hematology-Oncology Headlines)
Source: Medscape Hematology-Oncology Headlines - June 14, 2022 Category: Cancer & Oncology Tags: Cardiology News Alert Source Type: news

FDA Approves Amvuttra (vutrisiran) for the Treatment of the Polyneuropathy of Hereditary Transthyretin-Mediated Amyloidosis in Adults
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Jun. 13, 2022-- Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced that the U.S. Food and Drug Administration (FDA) approved Amvuttra (vutrisiran), an RNAi... (Source: Drugs.com - New Drug Approvals)
Source: Drugs.com - New Drug Approvals - June 13, 2022 Category: Drugs & Pharmacology Source Type: news

Janssen Presents Updated Data at EHA for Teclistamab in Patients with Relapsed or Refractory Multiple Myeloma
June 10, 2022 (VIENNA) – The Janssen Pharmaceutical Companies of Johnson & Johnson announced updated efficacy and safety results from the teclistamab cohort of the Phase 1b TriMM-2 study (NCT04108195). Teclistamab, an investigational, off-the-shelf, T-cell redirecting bispecific antibody targeting B-cell maturation antigen (BCMA) is being studied in combination with DARZALEX FASPRO® (daratumumab and hyaluronidase-fihj) in patients with relapsed or refractory multiple myeloma (RRMM) who have received three or more prior lines of therapy.1 Patients in the study, including a high proportion with prior anti-CD38 exposur...
Source: Johnson and Johnson - June 10, 2022 Category: Pharmaceuticals Tags: Innovation Source Type: news

Janssen Presents Updated Results Evaluating First-in-Class GPRC5D Bispecific Antibody Talquetamab in Heavily Pretreated Patients with Multiple Myeloma
June 10, 2022 (VIENNA) – The Janssen Pharmaceutical Companies of Johnson & Johnson announced updated results from the Phase 1 MonumenTAL-1 first-in-human dose-escalation study of talquetamab (NCT03399799), an investigational, off-the-shelf, T cell redirecting bispecific antibody targeting both GPRC5D, a novel multiple myeloma target, and CD3 on T cells.1 Results from the study showed encouraging responses in heavily pretreated patients with relapsed or refractory multiple myeloma (RRMM) who received talquetamab at the recommended subcutaneous Phase 2 dose (RP2D) administered weekly (QW) or every two weeks (Q2W).2 The...
Source: Johnson and Johnson - June 10, 2022 Category: Pharmaceuticals Tags: Innovation Source Type: news

Updated Data for Janssen ’s Bispecific Teclistamab Suggest Continued Deep and Durable Responses in the Treatment of Patients with Relapsed or Refractory Multiple Myeloma
CHICAGO, ILLINOIS, June 5, 2022 – The Janssen Pharmaceutical Companies of Johnson & Johnson announced updated efficacy and safety results from the teclistamab Phase 1/2 MajesTEC-1 study. Teclistamab is an investigational, off-the-shelf, T-cell redirecting bispecific antibody targeting B-cell maturation antigen (BCMA), which is being studied in patients with relapsed or refractory multiple myeloma (RRMM) who have received three or more prior lines of therapy.1 The data were featured as part of an oral session during the 2022 American Society of Clinical Oncology (ASCO) Annual Meeting. Additional poster presentations f...
Source: Johnson and Johnson - June 5, 2022 Category: Pharmaceuticals Tags: Innovation Source Type: news

Janssen to Highlight Science, Innovation and Advances in Robust Oncology Portfolio and Pipeline Through More Than 60 Data Presentations at ASCO and EHA
RARITAN, N.J., May 31, 2022 — The Janssen Pharmaceutical Companies of Johnson & Johnson announced today that new research and data from its robust oncology portfolio and pipeline of investigational therapies will be presented at the 2022 American Society of Clinical Oncology (ASCO) Annual Meeting, taking place June 3-7 in Chicago, and the European Hematology Association (EHA) 2022 Congress taking place in Vienna, Austria, June 9-12. More than 60 presentations from company-sponsored studies across the two congresses, including 14 oral presentations, will feature new data and updates for both approved and investigation...
Source: Johnson and Johnson - May 31, 2022 Category: Pharmaceuticals Source Type: news

Race May Impact Accuracy of Models Predicting Brain Amyloidosis
MONDAY, April 25, 2022 -- Some models for predicting brain amyloidosis are affected by race and may not provide consistent results across racial groups, according to a study published online April 21 in Neurology. Suzanne E. Schindler, M.D., Ph.D.,... (Source: Drugs.com - Pharma News)
Source: Drugs.com - Pharma News - April 25, 2022 Category: Pharmaceuticals Source Type: news

New Tool Detects Difficult-to-Diagnose Cardiac Conditions New Tool Detects Difficult-to-Diagnose Cardiac Conditions
A new algorithm has been developed that can reliably detect two cardiovascular conditions -- hypertrophic cardiomyopathy and cardiac amyloidosis -- that are often difficult to diagnose.Medscape Medical News (Source: Medscape Cardiology Headlines)
Source: Medscape Cardiology Headlines - March 17, 2022 Category: Cardiology Tags: Cardiology News Source Type: news

TRAPS-Related AA Amyloidosis TRAPS-Related AA Amyloidosis
This paper describes the frequency and prognosis of AA amyloidosis in TNF receptor-1-associated periodic syndrome. What is known about this potentially severe complication?Rheumatology (Source: Medscape Transplantation Headlines)
Source: Medscape Transplantation Headlines - January 7, 2022 Category: Transplant Surgery Tags: Rheumatology Journal Article Source Type: news

Ionis and AstraZeneca close deal to develop and commercialize eplontersen
CARLSBAD, Calif., Dec. 29, 2021 -- (Healthcare Sales & Marketing Network) -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS), the leader in RNA-targeted therapies, today announced the closing of the collaboration agreement with AstraZeneca to develop and comme... Biopharmaceuticals Ionis Pharmaceuticals, AstraZeneca, eplontersen, transthyretin amyloidosis (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - December 29, 2021 Category: Pharmaceuticals Source Type: news

New Data from MajesTEC-1 Study Show Continued Deep and Durable Responses of Teclistamab (BCMAxCD3 Bispecific Antibody) in Treatment of Heavily Pretreated Patients with Multiple Myeloma
The objective responsive rate observed in this study suggests a potential benefit for many patients with triple-class exposed disease with an off-the-shelf therapy.”As of September 2021, 165 patients were treated with teclistamab at the SC 1.5 mg/kg dose across both Phase 1 and Phase 2 of MajesTEC-1.1 The primary objectives of the MajesTEC-1 Phase 1 study (NCT03145181) were to identify the recommended SC RP2D (part 1) and characterize the safety and tolerability of teclistamab at the RP2D (part 2). The primary objective of the MajesTEC-1 Phase 2 study (NCT04557098) was to evaluate the efficacy of teclistamab at the RP2D,...
Source: Johnson and Johnson - December 13, 2021 Category: Pharmaceuticals Tags: Innovation Source Type: news

Janssen Presents Updated Results Evaluating First-in-Class Talquetamab (GPRC5DxCD3 Bispecific Antibody) in Heavily Pretreated Patients with Multiple Myeloma
ATLANTA, Ga., December 11, 2021– The Janssen Pharmaceutical Companies of Johnson & Johnson announced today updated results from the MonumenTAL-1 Phase 1 first-in-human dose-escalation study of talquetamab (NCT03399799). Talquetamab is the only investigational off-the-shelf T cell redirecting bispecific antibody in clinical development targeting both GPRC5D, a novel multiple myeloma target, and CD3 on T cells.[1] Results from the study show that no new safety signals were observed with longer follow-up.1 Heavily pretreated patients with multiple myeloma treated with talquetamab at the recommended subcutaneous (SC) Pha...
Source: Johnson and Johnson - December 11, 2021 Category: Pharmaceuticals Tags: Innovation Source Type: news

Janssen to Present the Strength and Promise of its Hematologic Malignancies Portfolio and Pipeline at ASH 2021
RARITAN, N.J., November 4, 2021 – The Janssen Pharmaceutical Companies of Johnson & Johnson announced today that more than 45 company-sponsored abstracts, including 11 oral presentations, plus more than 35 investigator-initiated studies will be featured at the American Society of Hematology (ASH) Annual Meeting and Exposition. ASH is taking place at the Georgia World Congress Center in Atlanta and virtually from December 11-14, 2021.“We are committed to advancing the science and treatment of hematologic malignancies and look forward to presenting the latest research from our robust portfolio and pipeline during ASH...
Source: Johnson and Johnson - November 5, 2021 Category: Pharmaceuticals Tags: Innovation Source Type: news

Can TTR Assays Track Tafamidis Success in Cardiac Amyloidosis? Can TTR Assays Track Tafamidis Success in Cardiac Amyloidosis?
Pending a practical way to check whether costly tafamidis is working in individuals with TTR amyloid cardiopathy, transthyretin levels just might help as a surrogate efficacy endpoint, researchers propose.Medscape Medical News (Source: Medscape Cardiology Headlines)
Source: Medscape Cardiology Headlines - October 19, 2021 Category: Cardiology Tags: Cardiology News Source Type: news