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Stung by a clinical trial miss in Duchenne muscular dystrophy, the biotech now has promising, if early, data to back a gene therapy for a different rare neuromuscular disease MORE
Credit: RCSI Scientists have developed polypeptide-based materials that act as effective vectors for delivering gene therapies. The first-of-its-kind platform enables the vectors to be adapted to suit the specific gene therapy cargo. MORE
The 2 nd Gene Therapy Immunogenicity Summit is the industry’s definitive forum enabling you to better modulate, measure and predict immune response to your gene therapy candidate. Performances of biosafety analytical assays commonly used in gene therapy development. MORE
Nearly $20 billion was invested in biotechs developing cell-, gene- and tissue-based treatments in 2020, according to a new report from industry group ARM MORE
The inaugural Gene Therapy Medical Affairs Summit is dedicated to exploring the pivotal internal and external role that Medical Affairs plays in delivering gene therapies to patients. The post Gene Therapy Medical Affairs appeared first on. MORE
Three companies are testing their gene therapies in early clinical trials, with initial results due later this year. Others are in preclinical stages and aim to follow soon MORE
Rocket Pharma’s Danon disease treatment — key to the company's quiet rise over the past year — is the latest gene therapy to be put on hold by the agency. MORE
Five trial participants with diabetic macular edema who received Adverum Biotechnologies' treatment experienced side effects never before reported for an eye gene therapy MORE
An investigation by UniQure determined the company's hemophilia gene therapy was "highly unlikely" to have caused a study volunteer's liver cancer, clearing the way for the FDA's green light MORE
Significant setbacks for pace-setting programs from BioMarin and UniQure have renewed questions about gene therapy’s promise, and its safety MORE
With clinical holds lifting there has been more investment and clinical progress in the gene therapy for muscular disorders space than ever before, yet the consequences of toxicity are not a distant memory. The post Gene Therapy for Muscular Disorders appeared first on. MORE
Natalie Holles, who was appointed CEO of Audentes soon after Astellas acquired the gene therapy developer in 2019, has departed amid the company's integration MORE
Orchard Therapeutics’ gene therapy for rare childhood disease metachromatic leukodystrophy (MLD) Libmeldy has been rejected for NHS use by NICE in draft guidance. Orchard has already offered a confidential discount on the therapy. MORE
A longer-than-expected timeline for developing a new manufacturing process means a key study for Axovant's top gene therapy likely won't begin until 2022 MORE
Sarepta and Pfizer are advancing their experimental treatments into late-stage trials, while others are accelerating research in what's become one of gene therapy's most active fields MORE
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 MORE
Credit: The Ohio State University Wexner Medical Center A novel method of gene therapy is helping children born with a rare genetic disorder called AADC deficiency that causes severe physical and developmental disabilities. Biology Gene Therapy Medicine/Health Neurobiology Pediatrics Surgery MORE
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 MORE
Cell and gene therapies (CGTs) are poised to revolutionize the landscape of biologic drugs that treat diseases and offer hope in areas where there was previously none MORE
The FDA's decision not to approve Roctavian raises the question of why BioMarin was able to submit the hemophilia A therapy in the first place MORE
The planned factory is a strong sign of Biogen's interest in gene therapy, an area of research it has invested in through dealmaking over the past few years MORE
Founded by gene therapy pioneer Jude Samulski, AskBio boasts a pipeline of experimental gene therapies as well as its own manufacturing capabilities MORE
A version of the treatment made at commercial scale looks similar to what Sarepta used in early testing. But the findings are no longer the last step before an approval filing, as the company once hoped MORE
The treatment, for a progressive, often deadly brain disease, could soon become Bluebird's third approved product and one of only a handful of marketed gene therapies in the world MORE
The Swiss pharma is the third large drugmaker to partner with Harvard spinout Dyno Therapeutics, a small biotech aiming to improve gene therapy technology MORE
The pharma licensed a treatment for a severe type of age-related vision loss, building on past deals that gave it a foothold in gene therapy MORE
Gene therapy developers will need to address a series of setbacks that have shaded optimism in the sector's fast growth MORE
Zynteglo sales have been on hold since February, when a patient in a clinical trial of another, related Bluebird medicine developed leukemia MORE
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. MORE
Another gene therapy from Nightstar Therapeutics, which Biogen bought for $800 million in 2019, has failed in a key clinical study MORE
An unconventional strategy is behind the rise of Rocket, a New Jersey biotech whose executives boldly talk of turning into the "Genentech of gene therapy MORE
Taysha Gene Therapies to Host Manufacturing Day Taysha Gene Therapies to Host Manufacturing Day Virtual event on Tuesday, July 27, 2021, at 10:00 a.m. MORE
The collaboration with Capsigen hands Biogen rights to a technology that could support the development of gene therapies for central nervous system and neuromuscular disorders MORE
Katherine High and Jude Samulski, two well-known gene therapy researchers, were longtime collaborators and competitors. Now they're colleagues at one of the world's largest drugmakers MORE
The inaugural Gene Therapy Comparability Summit is the only industry dedicated meeting to ensuring your manufacturing processes are effective and comparable at every stage of development, supporting a seamless transition to the larger scales required for commercialization. MORE
The results are first to emerge from a late-stage test of a hemophilia B gene therapy, and show UniQure's treatment can restore clotting protein levels to mild or even near normal levels MORE
The setback for Bluebird's LentiGlobin comes amid a string of manufacturing-related delays for gene therapy developers MORE
Just two months after launching, Jaguar Gene Therapy has raised another $139 million for a preclinical pipeline aimed at gene-linked autism and diabetes MORE
Flagship Pioneering, the biotech incubator that created Moderna, is helping fund Ring Therapeutics, a startup hoping to disrupt gene therapy development through the use of a different viral vector MORE
Company initially withdrew a marketing authorisation application for the gene therapy last year MORE
The biotech has joined a group of investors to form a new gene therapy developer led by one of its longtime executives and backed with $107 million MORE
A five-year alliance with high-profile biotech ElevateBio is designed to get around a bottleneck that has held back the institution’s gene therapy work MORE
Treatments from Bluebird bio and CRISPR Therapeutics have shown considerable promise and could soon be nearing regulatory review. A full pipeline is growing behind them MORE
Far along in testing for a hemophilia treatment, BioMarin is expanding its gene therapy research into diseases of the central nervous system and aims to use some of the institute's technology MORE
Gene Therapy Related Topics 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.
Bio Pharma Dive
JULY 22, 2021
Three companies are testing their gene therapies in early clinical trials, with initial results due later this year. Others are in preclinical stages and aim to follow soon
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Bio Pharma Dive
JULY 28, 2021
Flagship Pioneering, the biotech incubator that created Moderna, is helping fund Ring Therapeutics, a startup hoping to disrupt gene therapy development through the use of a different viral vector
Bio Pharma Dive
JULY 23, 2021
Five trial participants with diabetic macular edema who received Adverum Biotechnologies' treatment experienced side effects never before reported for an eye gene therapy
Bio Pharma Dive
JANUARY 8, 2021
Gene therapy developers will need to address a series of setbacks that have shaded optimism in the sector's fast growth
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
Bio Pharma Dive
MAY 21, 2021
The treatment, for a progressive, often deadly brain disease, could soon become Bluebird's third approved product and one of only a handful of marketed gene therapies in the world
Bio Pharma Dive
JUNE 15, 2021
Another gene therapy from Nightstar Therapeutics, which Biogen bought for $800 million in 2019, has failed in a key clinical study
Bio Pharma Dive
OCTOBER 22, 2020
The biotech has joined a group of investors to form a new gene therapy developer led by one of its longtime executives and backed with $107 million
Bio Pharma Dive
JULY 1, 2021
A five-year alliance with high-profile biotech ElevateBio is designed to get around a bottleneck that has held back the institution’s gene therapy work
Bio Pharma Dive
JULY 9, 2021
Zynteglo sales have been on hold since February, when a patient in a clinical trial of another, related Bluebird medicine developed leukemia
Bio Pharma Dive
DECEMBER 2, 2020
The pharma licensed a treatment for a severe type of age-related vision loss, building on past deals that gave it a foothold in gene therapy
Bio Pharma Dive
MAY 11, 2021
The collaboration with Capsigen hands Biogen rights to a technology that could support the development of gene therapies for central nervous system and neuromuscular disorders
Bio Pharma Dive
APRIL 1, 2021
Natalie Holles, who was appointed CEO of Audentes soon after Astellas acquired the gene therapy developer in 2019, has departed amid the company's integration
Bio Pharma Dive
MARCH 11, 2021
An unconventional strategy is behind the rise of Rocket, a New Jersey biotech whose executives boldly talk of turning into the "Genentech of gene therapy
Bio Pharma Dive
OCTOBER 26, 2020
Founded by gene therapy pioneer Jude Samulski, AskBio boasts a pipeline of experimental gene therapies as well as its own manufacturing capabilities
Pharma Phorum
JANUARY 21, 2021
With clinical holds lifting there has been more investment and clinical progress in the gene therapy for muscular disorders space than ever before, yet the consequences of toxicity are not a distant memory. The post Gene Therapy for Muscular Disorders appeared first on.
Bio Pharma Dive
MARCH 19, 2021
Stung by a clinical trial miss in Duchenne muscular dystrophy, the biotech now has promising, if early, data to back a gene therapy for a different rare neuromuscular disease
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
Bio Pharma Dive
JULY 8, 2021
Treatments from Bluebird bio and CRISPR Therapeutics have shown considerable promise and could soon be nearing regulatory review. A full pipeline is growing behind them
Bio Pharma Dive
MAY 18, 2021
A version of the treatment made at commercial scale looks similar to what Sarepta used in early testing. But the findings are no longer the last step before an approval filing, as the company once hoped
Bio Pharma Dive
APRIL 28, 2021
Far along in testing for a hemophilia treatment, BioMarin is expanding its gene therapy research into diseases of the central nervous system and aims to use some of the institute's technology
Bio Pharma Dive
JANUARY 21, 2021
Katherine High and Jude Samulski, two well-known gene therapy researchers, were longtime collaborators and competitors. Now they're colleagues at one of the world's largest drugmakers
Scienmag
JUNE 9, 2021
Credit: RCSI Scientists have developed polypeptide-based materials that act as effective vectors for delivering gene therapies. The first-of-its-kind platform enables the vectors to be adapted to suit the specific gene therapy cargo.
Bio Pharma Dive
NOVEMBER 5, 2020
The setback for Bluebird's LentiGlobin comes amid a string of manufacturing-related delays for gene therapy developers
Scienmag
JULY 12, 2021
Credit: The Ohio State University Wexner Medical Center A novel method of gene therapy is helping children born with a rare genetic disorder called AADC deficiency that causes severe physical and developmental disabilities. Biology Gene Therapy Medicine/Health Neurobiology Pediatrics Surgery
Pharma Phorum
APRIL 16, 2021
The 2 nd Gene Therapy Immunogenicity Summit is the industry’s definitive forum enabling you to better modulate, measure and predict immune response to your gene therapy candidate. Performances of biosafety analytical assays commonly used in gene therapy development.
Bio Pharma Dive
JUNE 1, 2021
Cell and gene therapies (CGTs) are poised to revolutionize the landscape of biologic drugs that treat diseases and offer hope in areas where there was previously none
Bio Pharma Dive
APRIL 26, 2021
An investigation by UniQure determined the company's hemophilia gene therapy was "highly unlikely" to have caused a study volunteer's liver cancer, clearing the way for the FDA's green light
Bio Pharma Dive
OCTOBER 30, 2020
A longer-than-expected timeline for developing a new manufacturing process means a key study for Axovant's top gene therapy likely won't begin until 2022
Bio Pharma Dive
OCTOBER 14, 2020
The Swiss pharma is the third large drugmaker to partner with Harvard spinout Dyno Therapeutics, a small biotech aiming to improve gene therapy technology
Bio Pharma Dive
APRIL 13, 2021
Just two months after launching, Jaguar Gene Therapy has raised another $139 million for a preclinical pipeline aimed at gene-linked autism and diabetes
Bio Pharma Dive
NOVEMBER 19, 2020
The results are first to emerge from a late-stage test of a hemophilia B gene therapy, and show UniQure's treatment can restore clotting protein levels to mild or even near normal levels
Pharma Phorum
JULY 9, 2021
Orchard Therapeutics’ gene therapy for rare childhood disease metachromatic leukodystrophy (MLD) Libmeldy has been rejected for NHS use by NICE in draft guidance. Orchard has already offered a confidential discount on the therapy.
Bio Pharma Dive
MARCH 4, 2021
The planned factory is a strong sign of Biogen's interest in gene therapy, an area of research it has invested in through dealmaking over the past few years
Bio Pharma Dive
MAY 11, 2021
Rocket Pharma’s Danon disease treatment — key to the company's quiet rise over the past year — is the latest gene therapy to be put on hold by the agency.
Bio Pharma Dive
JANUARY 28, 2021
Significant setbacks for pace-setting programs from BioMarin and UniQure have renewed questions about gene therapy’s promise, and its safety
Pharma Times
JUNE 29, 2021
Company initially withdrew a marketing authorisation application for the gene therapy last year
Pharma Phorum
NOVEMBER 20, 2020
The inaugural Gene Therapy Medical Affairs Summit is dedicated to exploring the pivotal internal and external role that Medical Affairs plays in delivering gene therapies to patients. The post Gene Therapy Medical Affairs appeared first on.
Pharma Phorum
JANUARY 21, 2021
The inaugural Gene Therapy Comparability Summit is the only industry dedicated meeting to ensuring your manufacturing processes are effective and comparable at every stage of development, supporting a seamless transition to the larger scales required for commercialization.
BioTech 365
JULY 20, 2021
Taysha Gene Therapies to Host Manufacturing Day Taysha Gene Therapies to Host Manufacturing Day Virtual event on Tuesday, July 27, 2021, at 10:00 a.m.
Bio Pharma Dive
MARCH 16, 2021
Nearly $20 billion was invested in biotechs developing cell-, gene- and tissue-based treatments in 2020, according to a new report from industry group ARM
Bio Pharma Dive
AUGUST 20, 2020
The FDA's decision not to approve Roctavian raises the question of why BioMarin was able to submit the hemophilia A therapy in the first place
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