Bio Pharma Dive

CRISPR touts new results as fresh questions surround 'off-the-shelf' CAR-T

Bio Pharma Dive

The gene editing company aims to expand its trial and add a second dose to improve effectiveness. However, safety worries have emerged since the FDA ordered CRISPR's rival Allogene to suspend clinical work

Relay's targeted cancer drug could be safer than its competitors. Is it more effective?

Bio Pharma Dive

Initial study results for Relay's experimental treatment offer some support for the biotech's protein motion technology, but raise some questions, too

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Sarepta outlines final push for Duchenne gene therapy

Bio Pharma Dive

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

Merck advantage grows as FDA approves Keytruda for first-line cervical cancer

Bio Pharma Dive

The company's immunotherapy is the only drug of its type cleared for advanced cervical cancer. Others are being tested, however

Gene therapy developer gets chance at a comeback with Pfizer deal

Bio Pharma Dive

Voyager Therapeutics recently scrapped much of its pipeline and replaced top executives. Now, a Pfizer deal on new technology gives investors hope

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

After long wait, Editas reveals first data for CRISPR gene editing treatment

Bio Pharma Dive

Early study results offer some hopeful signs of efficacy, but the small number of patients treated and short follow-up limit firmer conclusions

Allogene cell therapy trials halted by FDA after safety finding

Bio Pharma Dive

Researchers found evidence of a "chromosomal abnormality" in one patient treated with Allogene's CAR-T cell therapy, spurring the clinical hold and an investigation

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Worrisome side effects lead Pfizer to narrow Duchenne gene therapy trial

Bio Pharma Dive

Outside trial data reviewers have attributed three cases of severe muscle weakness to Pfizer’s treatment, causing the company to change the design of its closely watched Phase 3 study

Sarepta embarks on late-stage clinical trial of Duchenne gene therapy

Bio Pharma Dive

Initiation of the Phase 3 trial is an important milestone for the biotech after earlier setbacks, as well as for patients with the inherited muscle disease

Merck says antiviral pill effective against COVID-19, lifting hopes for first oral drug

Bio Pharma Dive

The drugmaker, along with partner Ridgeback Biotherapeutics, plan to ask the FDA for emergency authorization "as soon as possible "

Drugs 255

AstraZeneca immunotherapy combo extends survival in liver cancer trial

Bio Pharma Dive

The results are a rare positive result for AstraZeneca's experimental drug tremelimumab, which has come up short in a handful of other studies and cancer types

Drugs 191

CDC panel clears way for Pfizer booster shots, but divided on broader use

Bio Pharma Dive

Agency advisers supported giving a third Pfizer vaccine dose to older adults and those with medical conditions. But in a break with the FDA, they voted against recommending it for younger people in high-risk jobs

After talks with FDA, a biotech prepares to submit its closely watched ALS drug

Bio Pharma Dive

Executives at Amylyx Pharmaceuticals said that based on the agency's recent feedback, they will ask for approval of AMX0035 — likely within the next six months

Drugs 282

eCOA clinical trials: A simple, cost-effective approach to study build

Bio Pharma Dive

This new data capture model gives clinical trial sponsors control over study build

5 FDA approval decisions to watch in the fourth quarter

Bio Pharma Dive

Regulators face key decisions on COVID-19 shots for children and boosters for Moderna's and J&J's vaccines. Other closely watched drugs for multiple myeloma and depression are under review, too

Fourth trial volunteer dies in Astellas gene therapy study

Bio Pharma Dive

The study, which was suspended following three deaths last year, had been restarted in February after Astellas lowered the treatment dose used

FDA advisers support booster dose of Pfizer vaccine in older or high-risk adults

Bio Pharma Dive

In a back-and-forth meeting Friday, the committee opposed clearing a third shot widely for use in people older than 16, but agreed the benefits outweighed the risks for adults over 65 or those at risk of severe COVID-19

Armed with half a billion dollars, a startup aims to change how brain drugs are made

Bio Pharma Dive

Neumora Therapeutics launched Thursday with a $100 million investment from Amgen and another $400 million from a long list of venture capital firms, including Arch, which is taking another shot at building a neuroscience company

Drugs 227

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

Takeda halts studies of touted sleep disorder drug over safety concern

Bio Pharma Dive

The drugmaker suspended dosing in two mid-stage studies of its experimental narcolepsy treatment after a "safety signal" emerged

Drugs 227

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

Pfizer, Merck launch large new trials of oral COVID-19 drugs

Bio Pharma Dive

Both companies are racing to prove their oral antivirals as treatments for COVID-19. Key clinical results could come later this year

Trials 285

Collins to step down as NIH head in transition for research agency

Bio Pharma Dive

After 12 years in the top job, the veteran director says a new scientist should assume leadership of the world's largest biomedical research institution

FDA advisers unanimously back Moderna booster shot for many Americans

Bio Pharma Dive

But the committee members remain opposed to broad use of boosters in healthy young adults, for whom the benefits of an additional shot are less clear

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Top FDA vaccine officials to leave agency as decision on COVID-19 boosters looms

Bio Pharma Dive

Marion Gruber and Phil Krause, two veteran vaccine reviewers, are unexpectedly leaving the agency at a critical time. The search for their replacements will begin "imminently," division head Peter Marks said in a letter

Theravance to lay off 75% of employees alongside larger restructuring

Bio Pharma Dive

The move, which will affect an estimated 270 positions, follows a review by the company's board, and comes just weeks after one of its most advanced drugs failed a key test

Drugs 264

J&J heats up RSV race with new vaccine data

Bio Pharma Dive

The company's candidate is one of two to enter late-stage testing for a respiratory virus that causes hundreds of thousands of hospitalizations a year

FDA panel endorses second shot for adults who received J&J vaccine

Bio Pharma Dive

Agency advisers unanimously supported offering a second dose of J&J's coronavirus vaccine to adults who previously received it, though guidance could change as regulators evaluate mixing boosters

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

How a busy year for biotech IPOs measures up at the three-quarter mark

Bio Pharma Dive

Drugmaker initial public offerings could break records set just a year ago. But valuations and stock performance have slipped from historic highs

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Novartis pushes further into gene therapy for the eye with deal for Swiss startup

Bio Pharma Dive

The large drugmaker has acquired Arctos Medical and its preclinical research into optogenetics, an area Novartis already showed interest in with its buyout of Vedere Bio last year

'This is the time to double down': Top Vertex executives defend research plan as pressure builds

Bio Pharma Dive

A series of setbacks has led to criticism of the storied biotech's bid to diversify. But Vertex intends to stay the course, its CEO and top scientist say

Moderna founder's next big play in RNA raises $440 million

Bio Pharma Dive

Just a few months out of stealth mode, Laronde and its "endless RNA" technology have caught the attention of well-known investors like Fidelity, T. Rowe Price Associates and Invus

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New data from Israel, Pfizer show potent effect from third coronavirus shot

Bio Pharma Dive

The much-anticipated data arrive two days before an important FDA advisory meeting to discuss authorization of a booster dose for Pfizer's coronavirus vaccine

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

Chinese drugmaker Clover says vaccine prevents COVID-19 in large trial

Bio Pharma Dive

Positive results from Clover's Phase 3 study could mean another coronavirus vaccine option for the many countries still grappling with short supplies

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Eisai, Biogen to test FDA by asking for approval of second Alzheimer's drug

Bio Pharma Dive

The Japanese pharma and Biogen partner is filing an application for accelerated OK of lecanemab, which works similarly to the companies' controversially cleared Aduhelm

Drugs 226

US to offer coronavirus boosters to all Americans in aggressive plan to counter delta's spread

Bio Pharma Dive

The Biden administration's call comes ahead of FDA authorization for additional doses in people who aren't immunocompromised, although agency head Janet Woodcock joined other officials in supporting the plan

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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

Moderna expands mRNA vaccine supply in deal with new biotech producer

Bio Pharma Dive

The company has previously said it could make up to three billion vaccine doses in 2022, depending on how booster shots are authorized

Departing FDA officials, WHO leaders argue against broad rollout of COVID-19 booster shots

Bio Pharma Dive

Marion Gruber and Philip Krause, two top FDA vaccine reviewers who are unexpectedly departing this fall, joined senior WHO officials in saying booster vaccine doses are not yet widely needed

Pfizer says coronavirus vaccine is safe and spurs immune response in children

Bio Pharma Dive

The results position Pfizer and BioNTech to seek clearance in children as young as five years old. But the companies haven't yet shared data on a rare heart inflammation associated with their shot that's a concern for regulators