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.
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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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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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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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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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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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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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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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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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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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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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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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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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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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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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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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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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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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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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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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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Operational risks with gene therapies should improve with time, exec says
Pharmaceutical Technology
FEBRUARY 27, 2024
Making therapies feasible from biological, regulatory, and commercial standpoints drives scalable genetic medicine.
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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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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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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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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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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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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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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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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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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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Amicus Therapeutics files patent for improved gene therapy vector for targeted protein expression
Pharmaceutical Technology
DECEMBER 22, 2023
Discover Amicus Therapeutics' groundbreaking gene therapy vectors and methods of use. Enhance protein expression and cellular targeting for effective treatment of genetic disorders. Explore the patent claim now!
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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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Moderna and Generation Bio partner for genetic medicines development
Pharmaceutical Technology
MARCH 24, 2023
Moderna has entered a strategic partnership with Generation Bio for the development of non-viral genetic medicines. Moderna’s biological and technical expertise will be combined with core technologies of the non-viral genetic medicine platform from Generation Bio.
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Innovation in immuno-oncology: Leading companies in gene therapy delivery using viral vectors
Pharmaceutical Technology
FEBRUARY 15, 2023
In the last three years alone, there have been over 633,000 patents filed and granted in the pharmaceutical industry, according to GlobalData’s report on Immuno-oncology in Pharmaceuticals: Gene therapy delivery using viral vectors.
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Astellas Sees Path Forward for Gene Therapy Despite Four Patient Deaths
BioSpace
NOVEMBER 15, 2023
The Japanese pharma contends that an analysis of the four deaths in its AT132 gene therapy clinical trial shows it is still viable as a potential treatment for a fatal, rare genetic disease.
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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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Cell and gene therapy manufacturing: the next generation of startups
Bio Pharma Dive
JUNE 20, 2023
Joining larger CDMOs like Lonza and Catalent, a new group of companies aims to capitalize on persistent bottlenecks in producing complex genetic treatments.
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Capsida Biotherapeutics and Prevail to develop CNS gene therapies
Pharmaceutical Technology
JANUARY 5, 2023
Capsida Biotherapeutics and Eli Lilly and Company ’s wholly owned subsidiary Prevail Therapeutics have announced a partnership for the development of non-invasive gene therapies for central nervous system (CNS) diseases. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. By Cytiva Thematic.
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Ginkgo adds gene editing tools in buyout of Feng Zhang-founded startup
Bio Pharma Dive
FEBRUARY 28, 2024
The acquisition of Proof Diagnostics, which was formed to develop a COVID-19 test, gives Ginkgo a library of novel enzymes that can be used to make genetic medicines.
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Italfarmaco’s Duvyzat Wins FDA Approval as First Nonsteroidal Treatment for All Genetic Variants of DMD
XTalks
MARCH 28, 2024
Duvyzat, a histone deacetylase (HDAC) inhibitor that works to reduce inflammation and muscle loss, is the first nonsteroidal drug approved to treat patients with all genetic variants of DMD. Progressive muscle weakness in the disease is caused by genetic mutations in the dystrophin gene that lead to a lack of functional dystrophin protein.
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