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The biotech still believes a speedy approval filing for the closely watched treatment is possible, but expects to have to wait for the results of the recently launched Phase 3 trial, CEO Doug Ingram said on a conference call 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
New approach to gene therapy can correct any disease-causing mutation within a gene UNIVERSITY PARK, Pa. — A new approach to gene editing using the CRISPR/Cas9 system bypasses disease-causing mutations in a gene, enabling treatment of genetic diseases linked to a single gene, such as cystic fibrosis, certain types of sickle cell anemia, and other […]. Medicine & Health Biology Cell Biology Gene Therapy Genetics Medicine/Health MORE
LA JOLLA—(October 13, 2021) For the past fifteen years, cancer researchers have been using DNA sequencing technology to identify the gene mutations that cause the different forms of cancer. Medicine & Health cancers commonly gene mutated reveal Salk scientists 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
Five trial participants with diabetic macular edema who received Adverum Biotechnologies' treatment experienced side effects never before reported for an eye gene therapy MORE
On the heels of a two-day FDA meeting in which experts debated the risks of gene therapy, the biotech said its early-stage PKU treatment was associated with cancer in mice MORE
Significant setbacks for pace-setting programs from BioMarin and UniQure have renewed questions about gene therapy’s promise, and its safety MORE
Promising news in the effort to develop drugs to treat obesity: University of Virginia scientists have identified 14 genes that can cause and three that can prevent weight gain. Biology discover Genes obesity scientists MORE
The IPO is several times larger than what CRISPR Therapeutics, Intellia and Editas raised several years ago, and ranks well ahead of a more recent offering from gene editing startup Beam 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
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
Founded by former Biogen executive Michael Ehlers and backed by Apple Tree Partners, Intergalactic aims to create non-viral gene therapies delivered via an advanced form of electroporation 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 value of the biotech's growing gene therapy portfolio has been "unrecognized" by investors, said CEO John Crowley, leading to a sudden shift for an initiative that began just three years ago MORE
Cutting edge’ is, for once, a truly apt description when it comes to gene editing – both because the field is pushing medicine into areas we might never have dreamed possible, and because these technologies involve literally cutting DNA at a specific point in the genome. 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
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
Jude Children’s Research Hospital scientists have developed a highly efficient method to address a major challenge in biology–identifying the genetic ‘switches’ that regulate gene expression. Biology Gene Therapy Genes Genetics Medicine/Health MORE
A panel convened by the regulator suggested ways to make gene therapy research safer, but struggled to propose broader recommendations for the field MORE
The study, which was suspended following three deaths last year, had been restarted in February after Astellas lowered the treatment dose used MORE
The companies aim to develop therapies for complement-driven diseases, a specialty of Apellis and relatively new research ground for gene editing 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
Through a deal with Precision Biosciences, the pharma aims to develop therapies for genetic disorders, including Duchenne muscular dystrophy MORE
Guide Therapeutics, the company Beam acquired, is working on ways to more efficiently deliver gene editing therapies into the body MORE
Researchers create novel CRISPR-based fly species as a new method of controlling gene drive spread Credit: Akbari lab, UC San Diego CRISPR-based technologies offer enormous potential to benefit human health and safety, from disease eradication to fortified food supplies. 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
A group of advisers convened by the regulator will spend Thursday and Friday weighing a range of safety worries with gene therapy, and how best to prevent them MORE
Results published in The New England Journal of Medicine offer the first clinical evidence that CRISPR gene editing inside the body can be safe and effective, a culmination of years of scientific research 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
Capitalizing on investor interest in gene editing, the high-profile startup has secured one of the richest biotech initial public offerings priced this year 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
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
For an upfront payment of $45 million, Takeda gains access to as many as eight programs from Poseida Therapeutics and a potential method of delivering gene therapies without the help of viruses 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
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 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
SEPTEMBER 3, 2021
A panel convened by the regulator suggested ways to make gene therapy research safer, but struggled to propose broader recommendations for the field
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Bio Pharma Dive
OCTOBER 12, 2021
For an upfront payment of $45 million, Takeda gains access to as many as eight programs from Poseida Therapeutics and a potential method of delivering gene therapies without the help of viruses
Bio Pharma Dive
OCTOBER 7, 2021
Founded by former Biogen executive Michael Ehlers and backed by Apple Tree Partners, Intergalactic aims to create non-viral gene therapies delivered via an advanced form of electroporation
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
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
SEPTEMBER 1, 2021
A group of advisers convened by the regulator will spend Thursday and Friday weighing a range of safety worries with gene therapy, and how best to prevent them
Bio Pharma Dive
SEPTEMBER 7, 2021
On the heels of a two-day FDA meeting in which experts debated the risks of gene therapy, the biotech said its early-stage PKU treatment was associated with cancer in mice
Scienmag
OCTOBER 1, 2021
Promising news in the effort to develop drugs to treat obesity: University of Virginia scientists have identified 14 genes that can cause and three that can prevent weight gain. Biology discover Genes obesity scientists
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
Bio Pharma Dive
JUNE 17, 2021
Capitalizing on investor interest in gene editing, the high-profile startup has secured one of the richest biotech initial public offerings priced this year
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
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
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
SEPTEMBER 29, 2021
The value of the biotech's growing gene therapy portfolio has been "unrecognized" by investors, said CEO John Crowley, leading to a sudden shift for an initiative that began just three years ago
Bio Pharma Dive
OCTOBER 11, 2021
The biotech still believes a speedy approval filing for the closely watched treatment is possible, but expects to have to wait for the results of the recently launched Phase 3 trial, CEO Doug Ingram said on a conference call
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
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
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
JUNE 26, 2021
Results published in The New England Journal of Medicine offer the first clinical evidence that CRISPR gene editing inside the body can be safe and effective, a culmination of years of scientific research
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
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
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
JULY 23, 2021
The IPO is several times larger than what CRISPR Therapeutics, Intellia and Editas raised several years ago, and ranks well ahead of a more recent offering from gene editing startup Beam
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
Scienmag
APRIL 21, 2021
New approach to gene therapy can correct any disease-causing mutation within a gene UNIVERSITY PARK, Pa. — A new approach to gene editing using the CRISPR/Cas9 system bypasses disease-causing mutations in a gene, enabling treatment of genetic diseases linked to a single gene, such as cystic fibrosis, certain types of sickle cell anemia, and other […]. Medicine & Health Biology Cell Biology Gene Therapy Genetics Medicine/Health
Bio Pharma Dive
SEPTEMBER 14, 2021
The study, which was suspended following three deaths last year, had been restarted in February after Astellas lowered the treatment dose used
Pharma Phorum
JANUARY 29, 2021
Cutting edge’ is, for once, a truly apt description when it comes to gene editing – both because the field is pushing medicine into areas we might never have dreamed possible, and because these technologies involve literally cutting DNA at a specific point in the genome.
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
Scienmag
JUNE 2, 2021
Researchers create novel CRISPR-based fly species as a new method of controlling gene drive spread Credit: Akbari lab, UC San Diego CRISPR-based technologies offer enormous potential to benefit human health and safety, from disease eradication to fortified food supplies.
Bio Pharma Dive
NOVEMBER 5, 2020
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
Scienmag
MAY 6, 2021
Jude Children’s Research Hospital scientists have developed a highly efficient method to address a major challenge in biology–identifying the genetic ‘switches’ that regulate gene expression. Biology Gene Therapy Genes Genetics Medicine/Health
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
NOVEMBER 5, 2020
The setback for Bluebird's LentiGlobin comes amid a string of manufacturing-related delays for gene therapy developers
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
NOVEMBER 20, 2020
Through a deal with Precision Biosciences, the pharma aims to develop therapies for genetic disorders, including Duchenne muscular dystrophy
Scienmag
OCTOBER 13, 2021
LA JOLLA—(October 13, 2021) For the past fifteen years, cancer researchers have been using DNA sequencing technology to identify the gene mutations that cause the different forms of cancer. Medicine & Health cancers commonly gene mutated reveal Salk scientists
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
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
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
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
FEBRUARY 23, 2021
Guide Therapeutics, the company Beam acquired, is working on ways to more efficiently deliver gene editing therapies into the body
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
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