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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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.
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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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.
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.
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.
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.
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.
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.
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.
Pharmaceutical Technology
SEPTEMBER 15, 2023
Cure Genetics has entered a partnership and licensing deal with Frametact to develop gene therapy for familial neurological ailments.
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.
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.
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.
Pharmaceutical Technology
JANUARY 24, 2024
NAYA’s acquisition includes Florida Biotechnologies’ AAV gene therapy for the treatment of a rare genetic disorder.
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.
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.
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.
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.
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.
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.
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.
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.,
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.
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.
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.
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.
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
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.
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.
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!
XTalks
JANUARY 4, 2024
Pfizer has kickstarted the new year with its first-ever gene therapy approval, awarded by Health Canada to the company’s Beqvez (fidanacogene elaparvovec) for the treatment of hemophilia B. There is a significant focus on developing gene therapies as longer-term solutions for the disease.
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.
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
Drug Discovery World
JULY 19, 2024
Dr Gonzalez-Aseguinolaza said: “This is a tremendous honour, and I am very pleased that the RFS Society recognises my dedication and work in the field of gene therapy. She then moved into rare diseases and has been working on rare hepatic diseases for the last two decades, with a focus on the development of advanced therapies.
BioPharma Reporter
JULY 16, 2024
A gene therapy being developed by Lexeo Therapeutics to treat cardiomyopathy caused by Friedreich ataxia, a rare neurodegenerative genetic disease, has achieved good interim results in a phase 1/2 trial.
XTalks
MARCH 21, 2024
The US Food and Drug Administration (FDA) has awarded approval to Orchard Therapeutics for its gene therapy Lenmeldy (atidarsagene autotemcel) for the treatment of children with pre-symptomatic late infantile, pre-symptomatic early juvenile or early symptomatic early juvenile metachromatic leukodystrophy (MLD).
Pharmaceutical Technology
FEBRUARY 27, 2024
Making therapies feasible from biological, regulatory, and commercial standpoints drives scalable genetic medicine.
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.
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.
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.
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.
Cloudbyz
JULY 31, 2024
This revolution is enabling the growth of innovative biomarker-based precision medicine and cell and gene therapy, transforming both clinical research and post-market care. This allows for the selection of patients who are most likely to respond to a particular therapy, optimizing treatment efficacy and minimizing adverse effects.
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.
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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