STAT+: Cell and gene therapy CEOs share survival strategies amid biotech slump
CARLSBAD, Calif. — Biotech CEOs leading cell and gene therapy companies spoke frankly at a…
Category: gene therapy
STAT+: Taysha Gene Therapies’ plan to tackle rare diseases is crumbling
In 2020, as biotech stocks surged amid the pandemic, a startup called Taysha Gene Therapies…
Gene therapy death was caused by an unknown risk of the virus used, study suggests
Last October, a 27-year-old man with Duchenne muscular dystrophy died after receiving a CRISPR-based treatment…
STAT+: Thermo Fisher just opened a $180 million plant. Executives already are talking about expanding.
Thermo Fisher Scientific’s giant plant to manufacture gene therapies for drug companies is barely a year old,…
STAT+: They carry a gene for ALS but aren’t sick. What does medical research owe them?
Nine months after her mom died of ALS, Jean Swidler walked into a doctor’s office…
STAT+: Dana-Farber deal highlights a surprising trend: New cancer centers are in vogue
Boston isn’t the only city slated for a new cancer center. Far from it. There’s…
STAT+: AstraZeneca bets that gene therapy’s workhorse will still deliver the goods
Seng Cheng has been working in gene therapy for nearly as long as gene therapy…
Listen: Racing for gene therapy, a pioneering approval, & startups in the lurch
What difference does a day make? Who gets to have gene therapy? And are biotech…
STAT+: With an age cutoff for a new gene therapy, families and doctors scramble to treat kids before their 6th birthday
In mid-July, Kristen Secrist hopped on a call with her 5-year-old son’s doctor, who had…
Listen: Biotech layoffs, eye drug drama, & gene therapy milestones
Can biotech startups survive a downturn? Is Covid-19 a good business? And when did ophthalmology…
STAT+: Gene therapy is in crisis. For nine hours, the field’s leading minds looked for a solution
WASHINGTON — Jennifer Puck has successfully treated 10 children with a gene therapy for a…
Listen: One Duchenne patient’s bittersweet hope for new treatment
Duchenne muscular dystrophy is a devastating disease and, until very recently, was one without much…
STAT+: BioMarin wins approval for gene therapy to treat hemophilia A
The Food and Drug Administration on Thursday approved a gene therapy to treat people with…
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STAT+: After promising early data, gene therapy trial for Huntington’s takes puzzling turn
Last year, after a string of high-profile trial failures, the Huntington’s disease community got positive…
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STAT+: After gene therapy deaths, Astellas brings in potentially safer treatment for muscle disorder
Astellas Pharma said Thursday it will license and develop a new gene therapy for a…
STAT+: In its first tough test, CRISPR epigenome editing cuts cholesterol levels in monkeys
A defanged form of CRISPR, which doesn’t slice or nick DNA, but rather alters the…
STAT+: FDA official offers insights into whether gene therapy makers could use common manufacturing process
WASHINGTON — A Food and Drug Administration official provided some insights this week into the…
Listen: The FTC v. Amgen, Sarepta’s future, & Galapagos’ turnaround
Does the FTC have it out for pharma? What does it take to make an…
STAT+: New Biden science agency ARPA-H launches first program, targeting bone regrowth
WASHINGTON — A multibillion-dollar science agency tasked with slashing through research bureaucracy will start its…
STAT+: A new startup looks to turn the workhorse of gene therapy against cancer
In 1980, Life and Time magazines introduced America to a new cancer wonder drug: interferon,…
STAT+: FDA’s Califf talks cell and gene therapies, abortion pill, Alzheimer’s drugs
WASHINGTON — Senators grilled FDA Commissioner Rob Califf Wednesday on everything from the agency’s stance…
Listen: Bancel v. Bernie, Sarepta’s FDA runaround, & Regeneron’s ever-growing blockbuster
What would Jonas Salk say if he were here right now? How do you know…
STAT+: Uniqure’s Huntington’s gene therapy lowers key protein in small trial
A gene therapy for Huntington’s disease made by the Dutch biotech firm Uniqure showed early…
STAT+: FDA’s vaccines and biologics chief talks Covid-19, 2022 priorities, and getting back to gene, cell therapy review
WASHINGTON — No part of the Food and Drug Administration has been challenged by the…
STAT+: Patients with rare blood disorder benefit from Bluebird gene therapy while denied access in Europe
People with severe beta-thalassemia who live in Europe have been denied access to Bluebird Bio’s…
MGH Researchers Discover How Amyloid Beta Forms in the Brain
Amyloid beta (Abeta) is a protein that is believed to be at the root of…
STAT+: What’s in a name? Spark Therapeutics sues Bluebird Bio over the use of the word ‘spark’
A promotional campaign has sparked a legal dispute between Spark Therapeutics and Bluebird Bio (BLUE)…
STAT+: Affinia Therapeutics raises $110 million for gene therapy treatments
Affinia Therapeutics, a Waltham-based biotech working on gene therapies to treat diseases using technology developed…
STAT+: Bluebird’s withdrawal of therapy from Germany could chill talks over gene therapy prices across Europe
When Bluebird Bio secured European approval for its gene therapy to treat beta thalassemia in…
STAT+: Uniqure investigation clears hemophilia B gene therapy as cause of patient’s liver cancer
Uniqure, the Dutch gene therapy company, said Monday that its one-time treatment for hemophilia B…
Bluebird suspends studies of sickle cell gene therapy following cancer diagnoses in two more treated patients
Bluebird Bio said Tuesday that it has suspended clinical trials involving its gene therapy for…
STAT+: Sarepta gene therapy for Duchenne muscular dystrophy stumbles with failed study outcome
Sarepta Therapeutics on Thursday announced mixed results from the first randomized clinical trial of its…
STAT Plus: In a shocking decision, FDA rejects BioMarin’s gene therapy for hemophilia
The Food and Drug Administration on Wednesday rejected a one-time therapy for hemophilia A, delaying…
STAT Plus: Recapping ASCO 2020, talking cancer therapy combinations, and looking to the future
Before it even started, ASCO 2020 was one for the books. Held virtually for the…
STAT Plus: Pfizer says efficacy of its Duchenne gene therapy outweighs side effects
Pfizer’s experimental gene therapy to treat Duchenne muscular dystrophy appeared to improve muscle function in…
STAT Plus: This biotech just bought three failed drugs. Here’s why
Biopharma companies don’t usually buy experimental drugs that don’t work. But that’s exactly what CODA…
7. Looking Ahead
Coronary artery disease is responsible for the deaths of 370,000 Americans annually. Fortunately, exciting advancements…
STAT Plus: Gene therapy pioneer says the field is behind — and that delivery technology is ‘embarrassing’
CAMBRIDGE, Mass. — Gene therapy pioneer Dr. James Wilson is disappointed by the progress in…