FDA clears Vertex, CRISPR to test gene-editing therapy in sickle cell patients

With a clinical hold dating back to May resolved, the companies said they plan to start a Phase 1/2 study of CTX001 in the U.S. by year's end.

By Jacob Bell • Oct. 10, 2018

Roche, Ionis focusing on eye disease through new deal

On the heels of their Huntington's disease collaboration, the companies are teaming up again — this time on a therapy targeting the immune system.

By Jacob Bell • Oct. 10, 2018

Explore the Trendline➔

Cell therapy

The continued emergence of CAR-T therapy has fueled research into next-generation approaches and new applications, such as its use in autoimmune diseases.

By BioPharma Dive staff

Arbutus' clinical challenges trigger stock slide

Shares in the biotech dropped by more than half this week on news of several hurdles in the clinic for its hepatitis B research. 

By Suzanne Elvidge • Oct. 10, 2018

J&J's top-seller Stelara looks set to expand into ulcerative colitis

New data show Stelara led to higher rates of clinical remission and response than placebo. An approval would further expand the biologic's market.

By Andrew Dunn • Oct. 9, 2018

Sarepta builds out gene therapy manufacturing with latest CDMO deal

The biotech is thinking ahead, inking partnerships in recent months with Paragon Bioservices and Brammer Bio to support its gene therapy pipeline.​

By Suzanne Elvidge • Oct. 9, 2018

Celgene showcases Otezla's potential in scalp psoriasis

Patients receiving treatment achieved significant improvements in moderate-to-severe scalp psoriasis compared to placebo, according to fresh Phase 3 data.

By Suzanne Elvidge • Oct. 9, 2018

Loxo, Blueprint drive forward with RET-targeting cancer therapies

No selective inhibitors of the oncogene are currently approved in the U.S. — something both biotechs hope to change with their respective therapies.

By Ned Pagliarulo • Oct. 8, 2018

Merck gets a shot in the arm with expanded label for Gardasil

The HPV vaccine is now also approved for adults aged 27 through 45 years old, widening the addressable market for Merck.

By Suzanne Elvidge • Oct. 8, 2018

Lilly sweetens its position in diabetes with positive 'twincretin' data

Researchers found LY3298176, which targets both GLP-1 and GIP, significantly outperformed placebo on measures of glucose control and weight loss.

By Suzanne Elvidge • Oct. 5, 2018

Roche's flu drug strongly beat placebo. But could one data miss be critical?

Baloxavir marboxil fell short of showing a statistical edge over Roche's older Tamiflu in the overall study population, although it did so in a subgroup. 

By Andrew Dunn • Oct. 4, 2018

Sarepta gene therapy for DMD takes another step forward

Updated results, including data from a fourth boy with Duchenne muscular dystrophy, help to firm up the prospects for the biotech's gene therapy.

By Ned Pagliarulo • Oct. 4, 2018

Gilead's Biktarvy matches GSK's Triumeq in Phase 3 study

Biktarvy is a key drug for Gilead in its battle with GSK for HIV market share. New Phase 3 data could help Gilead make its case. 

By Suzanne Elvidge • Oct. 4, 2018

Roche drug shows benefit in spinal muscular atrophy

Yet attention remains focused on a gene therapy in development by Novartis, which could reshape treatment of the genetic neuromuscular disorder.

By Ned Pagliarulo • Oct. 3, 2018

Seattle Genetics' Adcetris tops chemo in Phase 3 lymphoma trial

An unexpected hit on overall survival could be a boost for the biotech and partner Takeda as they seek to expand the market for Adcetris. 

By Suzanne Elvidge • Oct. 2, 2018

10 biotechs set to shake up the industry's top ranks

Analysts at Cowen picked 10 midsized biotechs that might be next to catch the market's eye as pipeline setbacks and missed financial targets have frustrated investors of larger drugmakers.

By Ned Pagliarulo , Andrew Dunn • Oct. 1, 2018

Alnylam puts its chips on speedy OK for rare disease drug

An early look at late-stage data showed the RNAi drug givosiran significantly cut levels of a key disease biomarker. But will that be enough for the FDA?

By Ned Pagliarulo • Sept. 28, 2018

Aldeyra has positive dry eye data — for now

Though its lead candidate beat vehicle in a Phase 2 study, Aldeyra's presentation of the results raised questions about the trial readout.

By Jacob Bell • Sept. 26, 2018

Takeda's dream of a first-line ALK inhibitor supported by late-stage data

Pfizer, Roche and Novartis all have first-line drugs for ALK-positive lung cancer, putting pressure on Takeda to keep up in the increasingly competitive market.

By Jacob Bell • Sept. 25, 2018

AstraZeneca's Imfinzi shows its strength in Stage 3 lung cancer

Results released at World Lung spell out the cancer immunotherapy's survival benefit, bolstering AstraZeneca's place in the earlier, locally advanced setting.

By Ned Pagliarulo • Sept. 25, 2018

Tecentriq sets the bar in small-cell lung cancer

Roche owns the first successful Phase 3 study of an immunotherapy in the less-common lung cancer type. But others are closing in.

By Ned Pagliarulo • Sept. 25, 2018

AstraZeneca heart study delivers mixed results for diabetes drug

The pharma hopes the results will help Farxiga compete with Eli Lilly and Boehringer Ingelheim's rival Jardiance, which has carved out a leading position on the strength of its heart claims.

By Andrew Dunn • Sept. 24, 2018

Vascepa's big heart benefit pumps up Amarin's value by $2B

Amarin's CEO called it "the single most significant advance in preventative cardiovascular drug therapy since the advent of statin therapy."

By Andrew Dunn • Sept. 24, 2018

One bottle of hope for one amazing kid

How Fisher Clinical Services, by Thermo Fisher Scientific worked with a drug company to create a special clinical trial to save a patient with late stage brain cancer.

Sept. 24, 2018

What cheap data means for understanding the patient experience in clinical trials

More nuanced, dynamic insights into patient experience can decrease the burden on patients by extrapolating from objective data sources and reducing the number of questionnaires being asked.

By Paul O'Donohoe Medidata Solutions Scientific Lead, eCOA and Mobile Health • Sept. 24, 2018

Narcan developer secures grant for counter to opioid weaponization

BARDA is concerned that repeat doses of naloxone would not be available in a large-scale terrorist attack involving weaponized opioids. So it's tapped Opiant to develop a long-acting opioid overdose agent. 

By Suzanne Elvidge • Sept. 21, 2018