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).
Ophthalmologists eye gene therapies for vision-threatening conditions as NGS facilitates precision diagnosis
AuroBlog - Aurous Healthcare Clinical Trials blog
SEPTEMBER 9, 2024
The convergence of gene therapies and clinical research is pushing the boundaries of what’s possible in ophthalmic care, offering hope for more effective treatments and potential cures for a range of vision-threatening conditions.
Gene Therapy 
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FDA approves first-ever gene therapy to be directly administered to the brain
BioPharma Reporter
NOVEMBER 19, 2024
PTC Therapeutics has received FDA approval for a gene therapy to treat AADC deficiency, a rare genetic disorder that impairs the normal function of neurons.
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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.
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Cyagen and Neurophth partner to develop gene therapy vectors
Pharmaceutical Technology
NOVEMBER 8, 2022
Cyagen and Neurophth Therapeutics have entered a strategic partnership to jointly develop next-generation AAV gene therapy vectors for specific kinds of genetic ophthalmic ailments. Additionally, Neurophth will oversee the clinical trials and marketing of gene therapy products developed leveraging the new AAV capsids of Cyagen.
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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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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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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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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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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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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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PTC gets FDA okay for first brain-delivered gene therapy
pharmaphorum
NOVEMBER 13, 2024
More than two years after its approval in Europe, PTC Therapeutics' gene therapy for rare genetic disorder AADC deficiency will soon be made available to patients in the US.The FDA has approved PTC's eladocagene exuparvovec – known as Upstaza in Europe – under the Kebilidi trade name in the US.
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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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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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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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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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Gene therapy startup emerges with green light for first-of-its-kind trial
Bio Pharma Dive
SEPTEMBER 12, 2024
Spun out of research at Nationwide Children’s Hospital, Vironexis claims it will be the first company to start a study testing a cancer drug delivered via the adeno-associated viruses commonly used in genetic medicines.
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Kebilidi FDA-Approved as First Brain-Delivered Gene Therapy for AADC Deficiency
XTalks
NOVEMBER 14, 2024
PTC Therapeutics has gained US Food and Drug Administration (FDA) approval for its new gene therapy, Kebilidi (eladocagene exuparvovec), for treating aromatic L-amino acid decarboxylase (AADC) deficiency.
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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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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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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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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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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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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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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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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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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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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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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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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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Gene Therapy Skysona Linked to 7 Cases of Blood Cancer
XTalks
OCTOBER 15, 2024
Seven cases of blood cancer have been identified in new trial data for bluebird bio’s gene therapy Skysona (elivaldogene autotemcel). The patients had received the autologous hematopoietic stem cell-based gene therapy as a treatment for early-stage cerebral adrenoleukodystrophy (CALD), a rare and fatal neurodegenerative disorder.
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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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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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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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Ultragenyx Pharmaceutical opens new gene therapy plant in US
Pharmaceutical Technology
JUNE 22, 2023
Ultragenyx Pharmaceutical has opened its new gene therapy manufacturing facility in Bedford, Massachusetts, US. The company will produce gene therapies in the new advanced facility, using its proprietary Pinnacle PCL (producer cell line) manufacturing platform.
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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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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 broken new boundaries in pricing, too.
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Rocket Pharmaceuticals’ gene therapy receives FDA RMAT designation
Pharmaceutical Technology
MAY 24, 2023
The RMAT designation programme is intended to accelerate the drug’s development and review processes for products, including gene therapies. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. Topic sponsors are not involved in the creation of editorial content.
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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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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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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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