5 questions facing gene therapy in 2022

Bio Pharma Dive

As record levels of money pour into gene therapy research, biotechs are under increased pressure to address issues related to safety, delivery and overlapping pipelines

Pfizer expands into gene editing with Beam research deal

Bio Pharma Dive

Pfizer's work with mRNA vaccines led it explore other applications of the technology, resulting in a multi-year partnership with the high-profile biotech Beam Therapeutics on gene editing treatments for rare diseases

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Gene Therapy and Pharmacokinetics

Camargo

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.

A high-profile gene therapy biotech takes aim at Huntington's

Bio Pharma Dive

Passage Bio and the University of Pennsylvania's Gene Therapy Program said they have begun preclinical research aimed at simultaneously developing multiple approaches to treating Huntington's

Modernizing Pharmaceutical Supply Chains

Speaker: Nimesh Patel, Gregory Demitrack, and Sarah Barnes Humphrey

Register for our upcoming webinar to better understand the state of the pharmaceutical supply chain. FourKites partnered with Accenture to survey supply chain executives working in pharmaceuticals, biotech, cell and gene therapy, medtech, and other related industries.

At FDA meeting, gene therapy experts wrestle with field's blindspots

Bio Pharma Dive

A panel convened by the regulator suggested ways to make gene therapy research safer, but struggled to propose broader recommendations for the field

Vertex aligns with another CRISPR gene editing startup

Bio Pharma Dive

A new alliance with Mammoth Biosciences marks the latest evidence of Vertex's interest in gene editing, which has already been the focus of multiple deals with emerging biotechs

Pfizer gene therapy research delayed by trial changes, safety questions

Bio Pharma Dive

Data readouts for Pfizer's hemophilia gene therapies are now expected later than previously forecast, while the company also works through modifying a trial of its Duchenne treatment

Moderna, teaming with a startup, wades further into gene editing

Bio Pharma Dive

The biotech has identified gene editing as a natural fit for the technology it uses to deliver its vaccines and drugs. A deal with Metagenomi, a startup backed by Bayer, will further those ambitions

Takeda takes aim at a biotech's gene therapy work

Bio Pharma Dive

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

Gene therapy for Fabry: early stages, promising results

Bio Pharma Dive

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

Avrobio stops work on rare disease gene therapy after unexpected study results

Bio Pharma Dive

The biotech's treatment was one of the furthest along in testing for Fabry, a rare inherited disease that's become a target for gene therapy developers

Beam, Apellis partner in deal to expand gene editing's reach

Bio Pharma Dive

The companies aim to develop therapies for complement-driven diseases, a specialty of Apellis and relatively new research ground for gene editing

Gene editing biotech Verve to raise $267M in large IPO

Bio Pharma Dive

Capitalizing on investor interest in gene editing, the high-profile startup has secured one of the richest biotech initial public offerings priced this year

Biogen gene therapy deal has yet to bear fruit

Bio Pharma Dive

Another gene therapy from Nightstar Therapeutics, which Biogen bought for $800 million in 2019, has failed in a key clinical study

Gene therapy for hemophilia: So close, yet so far away

Bio Pharma Dive

Significant setbacks for pace-setting programs from BioMarin and UniQure have renewed questions about gene therapy’s promise, and its safety

Record funding flowed into cell, gene therapy companies last year

Bio Pharma Dive

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

Years later, a first-of-its-kind treatment shows the power, and limits, of gene therapy

Bio Pharma Dive

Luxturna, approved four years ago as the first gene therapy for an inherited disease in the U.S., is improving sight and quality of life for several of the patients who received it

Intergalactic Therapeutics launches with $75M to build a new type of gene therapy

Bio Pharma Dive

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

J&J pushes further into eye gene therapy

Bio Pharma Dive

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

Bluebird's next gene therapy gets backing from European regulator

Bio Pharma Dive

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

What to expect at the FDA's two-day meeting on gene therapy safety

Bio Pharma Dive

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

Researchers pioneer new method to edit genes in human cells

Scienmag

SAN FRANCISCO, CA—January 5, 2021—Over the past decade, the CRISPR genome-editing system has revolutionized molecular biology, giving scientists the ability to alter genes inside living cells for research or medical applications.

Sarepta, continuing its gene therapy push, helps launch a startup

Bio Pharma Dive

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

Audentes CEO exits as Astellas rebrands a gene therapy buyout

Bio Pharma Dive

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

The delicate dance of developmental genes

Scienmag

We now know that proteins are not just the products of genes, but that they also interact with genes, influencing and regulating the rhythm of their expression. Chemistry AND Physics dance delicate developmental Genes

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Lilly, Arch join Deerfield in backing new gene therapy biotech Jaguar

Bio Pharma Dive

Just two months after launching, Jaguar Gene Therapy has raised another $139 million for a preclinical pipeline aimed at gene-linked autism and diabetes

FDA halts tests of BioMarin drug amid heightened focus on gene therapy safety

Bio Pharma Dive

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

Gene Network changes associated with cancer onset and progression identify new candidates for targeted gene therapy

Scienmag

Cancer chemotherapy has undergone a paradigm shift in recent years with traditional treatments like broad-spectrum cytotoxic agents being complemented or replaced by drugs that target specific genes believed to drive the onset and progression of the disease.

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Epilepsy research boosts case for new gene therapy for Dravet syndrome

Scienmag

Research from the University of Virginia School of Medicine suggests how a newly developed gene therapy can treat Dravet syndrome, a severe form of epilepsy, and potentially prolong survival for people with the condition. Biology boosts case Dravet epilepsy gene research syndrome therapy

Bluebird, winding down in Europe, withdraws another rare disease gene therapy

Bio Pharma Dive

The biotech won EU approval for two gene therapies, Zynteglo and Syksona, both of which it's now said it will pull from market after difficulties negotiating reimbursement

A biotech's eye gene therapy faces longer odds as serious side effects pile up

Bio Pharma Dive

Five trial participants with diabetic macular edema who received Adverum Biotechnologies' treatment experienced side effects never before reported for an eye gene therapy

Bluebird hit with another hold for sickle cell gene therapy

Bio Pharma Dive

Study enrollment and treatment of sickle cell patients under 18 will be paused for safety reasons, another hurdle in a series of setbacks for the company

Boehringer Ingelheim takes next step in development of cystic fibrosis gene therapy

Bio Pharma Dive

Gene therapy could be a solution for the remaining 10%, but the path forward is challenging Drugs from Vertex can effectively treat the lung disease in as much as 90% of patients.

Gene editing startup Metagenomi raises $175M to advance research pipeline

Bio Pharma Dive

The biotech raised the Series B round during "probably one of the worst biotech markets" emerging drugmakers have faced in some time, a Metagenomi executive told BioPharma Dive

The 'dovetail' partnership meant to turn Boston Children's into a hub for gene therapy startups

Bio Pharma Dive

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

How cells correctly choose active genes

Scienmag

It is essential for cells to control precisely which of the many genes of their genetic material they use. Biology Active cells choose correctly GenesThis is done in so-called transcription factories, molecular clusters in the nucleus.

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A biotech startup raises $117M to deliver gene therapies in a new way

Bio Pharma Dive

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

Intellia, with first results, delivers a 'landmark' for CRISPR gene editing

Bio Pharma Dive

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

Gene therapy for Duchenne muscular dystrophy: Nearing final tests

Bio Pharma Dive

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

PDUFA VII: Implications for Developers of Cell and Gene Therapies

Camargo

Many of the advancements included in PDUFA VII are driven in large part by an increase in the development of innovative products such as cell and gene therapies. The increase is designed to strengthen CBER’s capacity and capabilities for regulating cell and gene therapies.

Bypassing broken genes

Scienmag

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

Amicus, looking for a boost, sells its gene therapy work to a SPAC

Bio Pharma Dive

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