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.
Genetics Related Topics 
World Orphan Drug Congresses Europe vs. The US: Global Perspectives on Genetic Testing
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.
Genetics 
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FDA clears AstraZeneca, Merck drug to treat some genetic breast cancers early
Bio Pharma Dive
MARCH 14, 2022
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.
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A new startup gets Takeda's backing to take complex genetic medicines 'off the shelf'
Bio Pharma Dive
FEBRUARY 11, 2021
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 pays Seattle Genetics $1.7B as antibody cancer drugs continue comeback
Bio Pharma Dive
SEPTEMBER 14, 2020
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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Lilly and ProQR to expand genetic medicine development agreement
Pharmaceutical Technology
DECEMBER 23, 2022
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 secures $1.25bn for genetic therapy development
Pharmaceutical Technology
JANUARY 19, 2024
BridgeBio Pharma has secured strategic financing of $1.25bn from Blue Owl Capital and CPP Investments for genetic therapies.
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Scribe and Sanofi expand genetic therapy development deal
Pharmaceutical Technology
JULY 18, 2023
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 and Primordial Genetics merge to form biotech company Primrose Bio
Pharmaceutical Technology
SEPTEMBER 19, 2023
Pelican Technology has combined business with Primordial Genetics under a deal to create a private biotechnology company Primrose Bio.
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Prasad’s FDA appointment pressures cell and gene therapy stocks
Bio Pharma Dive
MAY 6, 2025
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 and Frametact seek gene cures for neurological ailments
Pharmaceutical Technology
SEPTEMBER 15, 2023
Cure Genetics has entered a partnership and licensing deal with Frametact to develop gene therapy for familial neurological ailments.
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Scientists Discover a Genetic Variant That Seems to Limit HIV Infection
AuroBlog - Aurous Healthcare Clinical Trials blog
AUGUST 13, 2023
Now an international team of researchers has discovered a new genetic variant in people of African ancestries that appears to restrict HIV replication after an infection sets in. A tiny fraction of people are naturally resistant to HIV infections, and scientists want to understand why.
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We’ve Discovered A Subtle Genetic Imbalance That May Drive Aging
AuroBlog - Aurous Healthcare Clinical Trials blog
DECEMBER 21, 2022
Scientists have found an extremely subtle twist in the genetics of aging cells, one that seems to make them increasingly less functional as time goes on.
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Genetic ancestry and parental smoking linked to new genetic changes in children
Medical Xpress
MAY 17, 2025
Ancestry and lifestyle choices of parents may affect the rate and type of new genetic changes that arise in their children, new research has found.
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Leveraging Genetic Testing for Enrolling Rare Disease Trials
Worldwide Clinical Trials
JANUARY 24, 2024
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.
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Neurocrine Biosciences’ Crenessity Approved as First New Treatment in Decades for Rare Genetic Disorder CAH
XTalks
DECEMBER 18, 2024
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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Genetic diagnostics promises paradigm shift towards personalized, proactive healthcare
AuroBlog - Aurous Healthcare Clinical Trials blog
FEBRUARY 18, 2024
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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Biology opens up new frontiers in medicine to cure genetic diseases: Kiran Mazumdar Shaw
AuroBlog - Aurous Healthcare Clinical Trials blog
SEPTEMBER 17, 2024
We are witnessing a revolution in healthcare, driven by advances in genetics, Omics, RNA and CRISPR gene-editing technology, to deliver precision and personalised medicine, said Kiran Mazumdar-Shaw, executive chairperson, Biocon and Biocon Biologics. This holds the potential to cure genetic diseases that have plagued families for generations.
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GSK gives Wave a lift with genetic medicine deal
Bio Pharma Dive
DECEMBER 14, 2022
A four-year agreement between the companies comes with $170 million in cash and equity for Wave, which in recent years has dealt with clinical setbacks.
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Exsilio debuts with $82M, pitching a ‘leap’ forward for genetic medicine
Bio Pharma Dive
JUNE 25, 2024
The company claims it can address some of the field’s limitations with medicines delivered via messenger RNA and lipid nanoparticles.
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Lilly pours more money into genetic treatments for neurological diseases
Bio Pharma Dive
JANUARY 6, 2022
For $50 million, Lilly has secured rights to a technology that's meant to transport nucleic acid therapies in a way that's more targeted and better tolerated than other methods.
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AuroBlog - Aurous Healthcare Clinical Trials blog
NOVEMBER 14, 2023
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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Lilly pays $880M for Prevail, pushing further into genetic medicine
Bio Pharma Dive
DECEMBER 15, 2020
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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An AstraZeneca, Merck drug slows the return of genetic breast cancer. Will testing speed up?
Bio Pharma Dive
JUNE 3, 2021
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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FDA approves Regeneron drug for rare, genetic form of high cholesterol
Bio Pharma Dive
FEBRUARY 12, 2021
The biotech set an average annual price of $450,000 for the first-of-its-kind drug, which treats a condition that affects about 1,300 people in the U.S.
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Seattle Genetics stays step ahead of rivals with bladder cancer data
Bio Pharma Dive
SEPTEMBER 18, 2020
Data confirming the company's drug Padcev can extend the lives of bladder cancer patients should strengthen its position with regulators, although Immunomedics is close behind with a competing treatment.
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Study reveals genetic variants are more common in Parkinson’s disease patients
Pharma Times
AUGUST 9, 2024
The study revealed that 13% of patients have a genetic form of the neurological disease
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AI startup Basecamp allies with the Broad to dream up ‘programmable’ genetic medicines
Bio Pharma Dive
OCTOBER 9, 2024
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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Entering the era of prenatal gene therapies
Pharmaceutical Technology
MAY 2, 2025
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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Ionis, AstraZeneca change a key trial, fueling more questions about a genetic heart disease
Bio Pharma Dive
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.
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Regeneron ups genetic medicine investment with Decibel buyout
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.
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Rare Disease Day 2025: Rally for Rare Event Recap
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, (..)
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Bayer, Amgen back ReCode's plan to broaden genetic medicine's reach
Bio Pharma Dive
JUNE 29, 2022
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 and Sarepta link for rare genetic disease treatments
Pharmaceutical Technology
NOVEMBER 27, 2024
Arrowhead has entered a worldwide licensing and partnership agreement with Sarepta Therapeutics for rare genetic disease treatments.
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Ocuphire and Opus Genetics merge to develop IRD gene therapy
Pharmaceutical Technology
OCTOBER 23, 2024
Ocuphire Pharma has announced the acquisition of Opus Genetics to develop gene therapies for inherited retinal diseases (IRDs).
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‘In vivo’ cell therapy: expanding beyond CAR-T
Bio Pharma Dive
OCTOBER 18, 2022
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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Regeneron, in search of an eye gene therapy, turns to a young biotech
Bio Pharma Dive
APRIL 6, 2022
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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Biohaven stock slides on withdrawal of European marketing application
Bio Pharma Dive
APRIL 25, 2025
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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Merck wins approval for cancer drug acquired in 2019 biotech buyout
Bio Pharma Dive
AUGUST 16, 2021
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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Tome Biosciences debuts with $213M and a new way to edit the genome
Bio Pharma Dive
DECEMBER 12, 2023
Based on the work of MIT scientists, the well-funded startup is developing ways to insert large sizes of genetic material anywhere in the genome without damaging or breaking DNA.
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Verve partners with Lilly on gene editing therapy for heart disease
Bio Pharma Dive
JUNE 15, 2023
Lilly will pay the genetic medicine specialist $60 million upfront to work together on a treatment targeting a risk factor known as Lp(a).
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GUARDIAN Study Reveals How Genomic Sequencing Could Bridge Gaps in Newborn Screening
XTalks
OCTOBER 29, 2024
Genomic sequencing is a window into our genetic makeup. The GUARDIAN (Genomic Uniform-screening Against Rare Diseases In All Newborns) study, recently published in the Journal of the American Medical Association (JAMA) , highlights how adding genome sequencing to newborn screening could reshape early detection for rare genetic conditions.
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Arbor raises $74M amid genetic medicine’s funding slump
Bio Pharma Dive
MARCH 18, 2025
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.
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Amid industry turbulence, Chroma raises fresh funding to edit the epigenome
Bio Pharma Dive
MARCH 1, 2023
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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