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.
Regeneron gene therapy improves hearing in two children
Bio Pharma Dive
MAY 8, 2024
The results add to an early but growing body of evidence suggesting gene therapy may improve outcomes in young kids with a genetic form of deafness.
Gene Therapy 
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A Healthy Lifestyle Could Offset Genetic Risk For Early Death by a Hopeful 62%
AuroBlog - Aurous Healthcare Clinical Trials blog
MAY 7, 2024
When research tells us that our grandparents’ diets can affect our health decades later, it can be hard to shake the feeling that genetics spells out the trajectory of our wellbeing. But how much of our health is actually determined by our genes, and what effect does our lifestyle have, for better or worse? Can […]
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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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Ginkgo grows its gene therapy offerings with StrideBio deal
Bio Pharma Dive
APRIL 5, 2023
The deal hands Ginkgo technology for discovering and engineering capsids — the outer shell that protects the helpful genetic material in gene therapies.
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AstraZeneca buys Pfizer’s early gene therapy work for up to $1B
Bio Pharma Dive
JULY 28, 2023
The British pharma is deepening its investment in genetic medicine through a deal for a group of Pfizer’s gene therapy delivery tools, while the New York giant cuts back.
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Breakthrough: Rare Gene Mutation Offers Clues to Preventing Type 1 Diabetes
AuroBlog - Aurous Healthcare Clinical Trials blog
APRIL 29, 2024
A unique genetic mutation in two siblings – that has never been seen in anyone else – has been discovered by UK researchers at the University of Exeter, pointing the way towards new treatment options for type 1 diabetes. The mutation is in the gene for a protein called programmed death-ligand 1 (PD-L1), and a […]
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15% of Parkinson’s cases linked to genetic factors, study saysÂ
Drug Discovery World
AUGUST 6, 2024
Centogene has announced data from its Rostock International Parkinson’s Disease (ROPAD) Study revealing the genetic factors and prevalence of Parkinson’s disease (PD). The findings from the study indicate that approximately 15% of PD-related cases are tied to genetic variants, with the majority being linked to LRRK2 and GBA1.
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Sickle cell pipeline narrows as gene therapy developers rethink research plans
Bio Pharma Dive
FEBRUARY 23, 2023
Graphite Bio and Sangamo are stopping work on their respective sickle cell gene therapies, while Intellia revealed partner Novartis ended development of its genetic treatment for the blood disease.
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Astellas to support development of Taysha’s gene therapy programmes
Pharmaceutical Technology
OCTOBER 25, 2022
Ast ellas Pharma has announced plans to make a strategic investment to back the development of Taysha Gene Therapies’ adeno-associated virus (AAV) development programmes for Rett syndrome and giant axonal neuropathy (GAN). Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.
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Yet another gene therapy developer turns to layoffs
Bio Pharma Dive
APRIL 1, 2022
Taysha Gene Therapies is narrowing its research efforts and cutting around 35% of its workforce, the latest in a series of layoffs announced by makers of genetic medicines.
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Gene Therapy and Pharmacokinetics
Camargo
JULY 27, 2021
How and When to Incorporate PK Design into Your Gene Therapy Development Plan. Gene therapy, which was in its infancy around 30 years ago, is now becoming a more prominent treatment method in many therapeutic areas, from personalized therapy to mass vaccinations against COVID-19. Gene Therapy Definition.
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Genetic causes of colorectal cancer revealed in new study
Drug Discovery World
AUGUST 12, 2024
A new study has provided the most comprehensive analysis to date of the genetic makeup of colorectal cancer (CRC), providing unique understanding of its response to treatment. They’ve also uncovered new CRC cancer sub-groups (categories of cancer with specific genetic characteristics that affect how cancer behaves and responds to treatment).
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Regeneron expands in gene editing with Mammoth deal
Bio Pharma Dive
APRIL 25, 2024
“With each passing year, we're more committed to becoming a serious player in the genetic medicine space,” a Regeneron executive said.
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Lilly agrees to acquire gene therapy developer Akouos for $610m
Pharmaceutical Technology
OCTOBER 19, 2022
Eli Lilly and Company has signed a definitive agreement for the acquisition of all outstanding shares of precision genetic medicine firm Akouos for a total deal value of up to nearly $610m or up to $15.50 Akouos focuses on developing adeno-associated viral gene therapies to treat inner ear conditions, including sensorineural hearing loss.
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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. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. The companies entered the initial agreement in September last year. By Cytiva Thematic.
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AavantiBio and Catalent Announce Partnership to Support Development and Manufacturing of Gene Therapies for Rare Genetic Diseases
Pharma Mirror
APRIL 28, 2021
The post AavantiBio and Catalent Announce Partnership to Support Development and Manufacturing of Gene Therapies for Rare Genetic Diseases appeared first on Pharma Mirror Magazine. and Europe. Catalent will further support process optimization and look to reduce material.
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Ginkgo BioWorks gets grant for AAV vector for treating NPC1 with specific gene sequence
Pharmaceutical Technology
APRIL 15, 2024
Discover the groundbreaking gene therapy patent by Ginkgo BioWorks Inc for treating NPC1 using AAV vectors. Learn how this innovative approach offers hope for rare genetic disorders like Neimann-Pick Disease Type C.
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For every million-dollar gene therapy, Peter Marks gets an email from FDA chief Robert Califf
Fierce Pharma
MAY 8, 2024
After Orchard Therapeutics’ gene therapy for a rare genetic disease was approved in March, Peter Marks, M.D., received a simple email from Commissioner Robert Califf, M.D. | After Orchard Therapeutics’ gene therapy for a rare genetic disease was approved in March, Peter Marks, M.D.,
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NAYA buys gene therapy company Florida Biotech for $20mÂ
Pharmaceutical Technology
JANUARY 24, 2024
NAYA’s acquisition includes Florida Biotechnologies’ AAV gene therapy for the treatment of a rare genetic disorder.
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Forge Biologics and Labcorp partner for gene therapies development
Pharmaceutical Technology
MAY 4, 2023
Forge Biologics and global life sciences company Labcorp have announced a strategic adeno-associated virus (AAV) gene therapy development and manufacturing collaboration. This will improve accessibility to services for AAV-mediated gene therapy programmes. Topic sponsors are not involved in the creation of editorial content.
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Epigenetic Editing Explodes on the Heels of Gene Editing Success
BioSpace
MARCH 24, 2024
Ubiquitous potential, possible safety advantages and the recent growth of cell and gene therapy are driving investment in a different type of genetic editing.
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Forge Biologics joins BGTC for new AAV gene therapies development
Pharmaceutical Technology
MAY 24, 2023
Forge Biologics has joined the public-private collaboration, the Bespoke Gene Therapy Consortium (BGTC), to expedite the development and manufacture of new AAV [adeno-associated virus] gene therapies to treat patients with rare diseases. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.
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Bluebird, with little fanfare, is first to bring a second gene therapy to market
Bio Pharma Dive
JULY 21, 2021
The approval of Skysona for a genetic brain disease is a milestone for one of gene therapy's pioneering companies, but isn't expected to turn Bluebird's financial fortunes around.
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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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The Significance of the MHRA Approval and Upcoming FDA Review of the First Gene Editing Treatment
Worldwide Clinical Trials
NOVEMBER 27, 2023
Casgevy, the commercial product formerly known as exa-cel, is administered by taking stem cells out of a patient’s bone marrow and editing a gene in the cells in a laboratory, with the modified cells then infused back into the patient after conditioning treatment to prepare the bone marrow. In June 2023, the U.S.
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Nanoscope preps filing for retinitis pigmentosa gene therapy
pharmaphorum
MARCH 26, 2024
Nanoscope Therapeutics is on the brink of filing for FDA approval of what could be the first gene therapy for incurable eye disease retinitis pigmentosa (RP) that can be used regardless of underlying genetic mutations.
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Lilly gene therapy finding puts focus on hearing loss treatment pipeline
Bio Pharma Dive
JANUARY 24, 2024
An 11-year-old boy born deaf can hear after receiving Lilly's genetic medicine. Others, including biotechs in China and Regeneron in the U.S., are taking aim at the same drug target.
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Pricey new gene therapies for sickle cell pose access test
Bio Pharma Dive
DECEMBER 8, 2023
million, while a competing genetic medicine also cleared Friday is priced at $3.1 Casgevy, the first CRISPR therapy approved by the FDA, will cost $2.2
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Beacon Therapeutics enters gene therapy arena with $120m launch
Pharmaceutical Technology
JUNE 13, 2023
Beacon Therapeutics has kickstarted its entry into the gene therapy field with a $120m Series A financing. Amongst it was AGTC’s lead clinical candidate, AFTC-501, an adeno-associated virus (AAV) gene therapy for XLRP. Beacon Therapeutics is the third gene therapy company Syncona has launched.
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Researchers identify potential new genetic target for cancer drugs
Drug Discovery World
JULY 25, 2024
UK researchers have mapped the exact variants in a gene that dramatically increase a person’s risk of developing cancer, paving the way for targeted treatment across diverse populations. The research team tested all 18,108 possible DNA changes in the BAP1 gene using ‘saturation genome editing’.
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Former AveXis executives launch gene therapy startup with uncommon targets
Bio Pharma Dive
FEBRUARY 25, 2021
Sean Nolan and Joe Nolan, former leaders at the Zolgensma developer, are behind Jaguar Gene Therapy, which plans to research treatments for a rare metabolic disease as well as genetically linked autism and diabetes.
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bluebird bio wins back-to-back landmark FDA approvals for first-in-class gene therapies
Pharmaceutical Technology
SEPTEMBER 30, 2022
After several setbacks, bluebird bio bounces back with two major FDA gene therapy approvals. Last month, Zynteglo (betibeglogene autotemcel), or beti-cel, was approved as a one-time potentially curative gene therapy for patients with beta-thalassaemia who require regular blood transfusions.
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Cell and gene therapies: why advanced medicines call for specialised logistics
Pharmaceutical Technology
JULY 7, 2022
Genetic mutations, both germline and acquired, are behind a large proportion of the most debilitating and sometimes life-threatening human diseases. Recent years have seen a steadily growing number of approvals for cell and gene therapies, which has spurred on the community to continue innovating in this space.
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Krystal Biotech receives FDA approval for DEB topical gene therapy
Pharmaceutical Technology
MAY 26, 2023
Krystal Biotech has received approval from the US Food and Drug Administration for topical gene therapy VYJUVEK to treat dystrophic epidermolysis bullosa (DEB) in adults and in children aged six months and above. VYJUVEK is designed to address the underlying genetic cause of the disease. It prevents epidermis adhesion to the dermis.
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ElevateBio raises funds to advance cell and gene therapies
Pharmaceutical Technology
MAY 25, 2023
ElevateBio has raised $401m in a Series D financing round for advancing its technology platforms to expedite the design, production and development of cell and gene therapies. The technology platforms include the Life Edit gene editing platform, an RNA, cell, protein, vector engineering and induced pluripotent stem cells (iPSCs) platform.
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Regeneron gene therapy restores hearing in profoundly deaf child
BioPharma Reporter
MAY 13, 2024
Regeneron Pharmaceuticalsâ gene therapy for otoferlin-related hearing loss, a genetic cause of deafness, has restored hearing to normal levels in one deaf child within just 24 weeks.
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Biologists create new genetic systems to neutralize gene drives
Scienmag
SEPTEMBER 18, 2020
Two active genetics strategies help address concerns about gene-drive releases into the wild Credit: Ana Silva In the past decade, researchers have engineered an array of new tools that control the balance of genetic inheritance.
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Orchard sets out to sell world’s priciest gene therapy
Bio Pharma Dive
MARCH 20, 2024
million list price, the highest of any genetic medicine to come to market. Orchard is counting on the long-term data it’s accrued to convince insurers to cover Lenmeldy’s $4.25
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Orchard sets new gene therapy price tag at $4.25M—the steepest of any drug in the US
Fierce Pharma
MARCH 20, 2024
After scoring an FDA nod Monday for the first gene therapy to treat the rare genetic disease metachromatic leukodystrophy (MLD) in the U.S., Kyowa Kirin and its subsidiary Orchard Therapeutics have | After scoring an FDA nod Monday for the first gene therapy to treat the rare genetic disease metachromatic leukodystrophy in the U.S.,
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Regeneron, Intellia target neurological diseases in expanded gene editing deal
Bio Pharma Dive
OCTOBER 3, 2023
The longtime partners believe that, by combining their technologies, they can create “in vivo” genetic medicines for nervous system and muscular disorders.
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3 Key Takeaways from Global Genes Summit 2023
Worldwide Clinical Trials
OCTOBER 24, 2023
Global Genes is a rare disease umbrella organization dedicated to eliminating the burdens and challenges of rare diseases for patients and families globally. Roughly 80% of rare diseases are due to a known genetic driver. Over the years, I have attended several Global Genes Summits.
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