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.
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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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). This would allow the companies to boost the development of new therapy options for Rett syndrome and GAN patients.
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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.
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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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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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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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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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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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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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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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NAYA buys gene therapy company Florida Biotech for $20m
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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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.
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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. ElevateBio intends to use the funds to advance its genetic medicine current good manufacturing practice (cGMP) and process development business, BaseCamp.
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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.
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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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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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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 broken new boundaries in pricing, too.
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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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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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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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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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Pricey new gene therapies for sickle cell pose access test
Bio Pharma Dive
DECEMBER 8, 2023
Casgevy, the first CRISPR therapy approved by the FDA, will cost $2.2 million, while a competing genetic medicine also cleared Friday is priced at $3.1
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Rocket Pharmaceuticals’ gene therapy receives FDA RMAT designation
Pharmaceutical Technology
MAY 24, 2023
It can correct several disease elements associated with PKD, a rare, monogenic red blood cell disorder caused by a mutation in the PKLR gene. It can correct several disease elements associated with PKD, a rare, monogenic red blood cell disorder caused by a mutation in the PKLR gene.
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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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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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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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FDA delays Sarepta’s DMD gene therapy decision until June
Pharmaceutical Technology
MAY 25, 2023
Pushing back an initial deadline, the US Food and Drug Administration (FDA) has proposed a new regulatory action date of 22 June, by which time the agency will assess the logistics of a possible approval for Sarepta Therapeutics’ Duchenne muscular dystrophy (DMD) gene therapy.
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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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Why demand is rising for secure and climate-controlled gene therapy services
Pharmaceutical Technology
JUNE 19, 2023
Although only a small number of gene therapies have reached the market thus far, the industry is poised to grow quickly over the next few years. According to GlobalData’s clinical trials database, there are currently 1,231 planned and ongoing trials for gene therapies and gene-modified cell therapies alone.
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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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FDA approves first gene therapy for Duchenne muscular dystrophy
Drug Discovery World
JUNE 23, 2023
Sarepta Therapeutics’ Elevidys has become the first gene therapy for Duchenne muscular dystrophy (DMD) to gain marketing authorisation in the US. It is contraindicated in patients with any deletion in exon 8 and/or exon 9 in the DMD gene.
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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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Opinion: The Transformative Potential of Gene Therapy for Neurodegenerative D.
BioSpace
DECEMBER 3, 2023
For forms of Alzheimer’s, frontotemporal dementia and Parkinson’s caused by genetic defects, gene therapy could change the treatment landscape.
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Bespoke Gene Therapy Consortium Selects 8 Rare Diseases for Clinical Trial Portfolio
XTalks
MAY 17, 2023
The Foundation for the National Institutes of Health (FNIH) announced this week that the Accelerating Medicines Partnership Bespoke Gene Therapy Consortium (AMP BGTC) has selected eight rare diseases for its clinical trial portfolio.
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Great Britain first to approve CRISPR-based gene therapy
Drug Discovery World
NOVEMBER 16, 2023
The Medicines and Healthcare products Regulatory Agency (MHRA) has authorised the world’s first gene therapy for sickle-cell disease (SCD) and transfusion-dependent β-thalassemia (TDT). Casgevy is designed to work by editing the faulty gene in a patient’s bone marrow stem cells so that the body produces functioning haemoglobin.
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Lyfgenia and Casgevy Become First FDA-Approved Gene Therapies for Sickle Cell Disease
XTalks
DECEMBER 13, 2023
The US Food and Drug Administration (FDA) has approved the first gene therapies for the treatment of sickle cell disease, approving two on the same day. Both gene therapies are approved for individuals 12 years of age and older with sickle cell disease. Both therapies will be available in early 2024.
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Gene therapy specialist bluebird exits “untenable” European market
pharmaphorum
AUGUST 10, 2021
Europe market has proved so hostile to gene therapies when it comes to pricing and reimbursement for gene therapies that bluebird bio has decided to quit the market altogether, according to Andrew Obenshain, president of its severe genetic diseases unit. market and will wind down in Europe.
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Ovid turns to gene therapy startup to restock drug pipeline
Bio Pharma Dive
AUGUST 24, 2022
The New York biotech will invest in and develop up to three drugs with Gensaic, an emerging startup aiming to use the viruses that infect bacteria to deliver genetic medicines.
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How can we fulfill the promise of Cell and Gene Therapy?
Pharma Mirror
JANUARY 30, 2022
Ben Beckley, Global Lead at EmerGENE explores the market challenges holding back Cell and Gene Therapy (C>) from taking its place as an established treatment area. There are several reasons for this growth, including high global cancer rates, The post How can we fulfill the promise of Cell and Gene Therapy?
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‘A watershed moment for the field of gene therapy’: bluebird bio’s gene therapy Zynteglo receives FDA approval
BioPharma Reporter
AUGUST 18, 2022
Food and Drug Administration (FDA) has approved bluebird bioâs Zynteglo (betibeglogene autotemcel), also known as beti-cel: a one-time gene therapy custom-designed to treat the underlying genetic cause of beta thalassemia in adult and pediatric patients who require regular red blood cell (RBC) transfusions.
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