FDA clears Ensoma’s application for rare genetic disorder treatment
Pharmaceutical Technology
MAY 13, 2025
The FDA has cleared the IND for Ensoma's EN-374, targeting X-linked chronic granulomatous disease (X-CGD), a rare genetic disorder.
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Pharmaceutical Technology
MAY 13, 2025
The FDA has cleared the IND for Ensoma's EN-374, targeting X-linked chronic granulomatous disease (X-CGD), a rare genetic disorder.
Worldwide Clinical Trials
DECEMBER 19, 2024
At the recent World Orphan Drug Congresses (WODC) in Europe and the United States, Worldwide Clinical Trials Derek Ansel , Vice President, Therapeutic Strategy Lead, Rare Disease, facilitated roundtable discussions to explore the operational challenges and ethical barriers surrounding genetic testing.
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Bio Pharma Dive
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Lynparza is the first drug targeting BRCA mutations to be approved for use in early breast cancer, a decision that could lead to more genetic testing.
Bio Pharma Dive
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Ensoma Therapeutics debuts with a lucrative alliance with the Japanese pharma and an unusual way to deliver genetic medicines into the body.
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Merck's deal for Seattle Genetics' antibody-drug conjugate comes as Gilead agreed to acquire rival developer Immunomedics in the biotech industry's largest buyout of 2020.
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Eli Lilly and Company has expanded a licencing and partnership agreement with ProQR Therapeutics to discover, develop and market new genetic medicines. The post Lilly and ProQR to expand genetic medicine development agreement appeared first on Pharmaceutical Technology.
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BridgeBio Pharma has secured strategic financing of $1.25bn from Blue Owl Capital and CPP Investments for genetic therapies.
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Scribe Therapeutics and Sanofi have expanded partnership to progress the development of in vivo genetic therapies to treat genomic diseases.
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Pelican Technology has combined business with Primordial Genetics under a deal to create a private biotechnology company Primrose Bio.
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A vocal opponent of his predecessor Peter Marks, Vinay Prasad will now lead the office tasked with reviewing some genetic medicines, adding more uncertainty to an already struggling field of research.
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Cure Genetics has entered a partnership and licensing deal with Frametact to develop gene therapy for familial neurological ailments.
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Written By: Derek Ansel, MS, CCRA, Executive Director, Therapeutic Strategy Lead, Rare Disease Given that 80% of rare diseases have a genetic etiology, genetic implications should be addressed at the onset of a clinical program to support trial enrollment. One diagnostic example that I discussed in my presentation is autism.
XTalks
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CAH is a group of rare genetic disorders affecting the adrenal glands, which produce essential hormones like cortisol, aldosterone and androgens. The most common form of CAH, 21-hydroxylase deficiency, disrupts hormone production, leading to cortisol deficiency and an overproduction of androgens.
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Indian healthcare industry is of the view that emergence of genetic diagnostics will be a paradigm shift towards personalized medicine. Through the ever-evolving landscape of healthcare, the role of genetic diagnostics offers personalized insights and proactive solutions for disease management and prevention.
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Indian healthcare looks at pharmacogenomics, the study of influence of genetic factors on drug response, in patient care as comprehensive genetic tests gain ground.
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The acquisition marks a significant bet by Lilly on gene therapy's potential to address tough-to-treat neurological disorders like Parkinson's disease.
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Study results disclosed ahead of ASCO could make Lynparza a standard "adjuvant" treatment for people with an inherited form of breast cancer — as long as they know they have it.
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AUGUST 9, 2024
The study revealed that 13% of patients have a genetic form of the neurological disease
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The company, which is building a database of biological interactions found in nature, revealed $60 million in funding and a collaboration with David Liu’s lab.
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As scientists explore treating genetic disorders before birth, the promise of foetal gene therapy is growingbut there are hurdles to overcome.
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MAY 2, 2022
The partners increased the size and length of a large, ongoing study in transthyretin amyloidosis cardiomyopathy, a decision that has implications Alnylam Pharmaceuticals, which is testing two of its drugs against the disease.
Bio Pharma Dive
AUGUST 9, 2023
Third Rock-founded Decibel, which has shed much of its value since a 2021 IPO, expected to run out of money within a year.
Worldwide Clinical Trials
APRIL 29, 2025
At our nearby venue at the Hyatt Regency Bethesda in Maryland, on February 28th, 2025, the presentations included: You Are Not Alone Michele Herndon, DNP, RN, Interim Director, Undiagnosed Disease Network Foundation Beyond DNA and Diagnosis: The Expanding Role of Genetic Counselors in Advocacy Ashley Thompson, MS, CGC, Founder and Principal Consultant, (..)
Bio Pharma Dive
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The two drugmakers joined a long list of investors, including Pfizer and Sanofi, that have been funding the startup’s plan to develop a new type of lipid nanoparticle technology.
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Arrowhead has entered a worldwide licensing and partnership agreement with Sarepta Therapeutics for rare genetic disease treatments.
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Ocuphire Pharma has announced the acquisition of Opus Genetics to develop gene therapies for inherited retinal diseases (IRDs).
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At least five startups have emerged with new ways to genetically modify immune cells within the body, an approach that, if successful, could widen the field of CAR-T treatment.
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Facing mounting competition to its top-selling drug Eylea, Regeneron is teaming up with ViGeneron to develop a genetic medicine for an inherited retinal disease.
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The drug, being developed for a rare genetic disorder and currently under review in the U.S., is seen by some investors as critical to Biohaven’s future.
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The FDA cleared Welireg, which Merck picked up in its $1 billion takeover of Peloton Therapeutics, to treat certain tumors associated with a rare genetic disease.
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Bio Pharma Dive
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XTalks
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The round will fund a “one-and-done” treatment for a rare kidney condition that its CEO says can address concerns patients have with therapies from Alnylam and Novo Nordisk.
Bio Pharma Dive
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At a time when many startups have struggled to gain cash, the biotech has raised another $135 million to back its research, a new twist on genetic medicine.
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