Tome acquires startup Replace, gaining new genome editing tools

The deal is the second startup sale engineered by University of California, Berkeley scientist Shakked Halperin, and gives Tome a way to insert or delete small DNA sequences into the genome.

By Ben Fidler • Jan. 2, 2024

Ionis, Astrazeneca win FDA approval of competitor to Alnylam drug

The clearance of Wainua for transthyretin amyloidosis opens up a new front in a long-running commercial battle between Ionis and Alnylam. 

By Ben Fidler • Dec. 22, 2023

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

Clene says its ALS drug data is not yet enough for FDA

The agency said initial trial evidence wouldn’t support an accelerated approval, according to the company, which plans to provide more data next year.

By Jacob Bell • Dec. 21, 2023

J&J bets bigger on MeiraGTx’s eye gene therapy

Ahead of a Phase 3 readout that could come next year, the pharma is paying $130 million in upfront and near-term cash for rights to the retinitis pigmentosa treatment that it didn’t already own.

By Jonathan Gardner • Dec. 21, 2023

BioMarin strikes deal with Elliott, agrees to review business

Under pressure from the activist investor, BioMarin will add more independent directors and set up an operational review committee.

By Kristin Jensen • Dec. 21, 2023

Argenx autoimmune drug study fails in blow to expansion hopes

The Dutch biotech’s star product has lost some luster after a second failed Phase 3 trial in two months, this time for a skin condition called pemphigus.

By Kristin Jensen • Dec. 20, 2023

Biogen holds onto Tecfidera market in Europe for a little longer

A decision by European regulators to revoke approvals for five generic versions of Biogen's multiple sclerosis medicine helps cement the drug's monopoly there through early 2025.

By Ned Pagliarulo • Dec. 19, 2023

Point radiopharma drug results disappoint in prostate cancer study

While the study met its goal, the drug’s benefit was less than analysts had predicted, an outcome that could hasten a planned takeover by Eli Lilly.

By Ned Pagliarulo • Dec. 18, 2023

Structure shares sink as obesity pill misses expectations in small study

Results from a Phase 2 trial fell short of what’s been seen with a similar Eli Lilly medicine, causing the closely watched biotech’s shares to plummet.

By Ben Fidler • Dec. 18, 2023

Pivotal Life Sciences reloads with $389M, hunting for deals in a downturn

The firm is tracking startups that launched during the biotechnology sector’s peak a few years ago but now need fresh funds to move their programs forward, one of its investors said.

By Gwendolyn Wu • Dec. 18, 2023

European regulators push Biogen, CRISPR drugs toward approval

While a positive recommendation for the gene editing treatment Casgevy was expected, a clearance for Biogen’s Skyclarys has been no sure bet.

By Ben Fidler • Dec. 15, 2023

Vir to lay off staff, cut costs amid setbacks building on COVID success

The biotech is letting go of 75 employees and closing two R&D facilities, continuing a strategic reset under new CEO Marianne de Backer. 

By Delilah Alvarado • Dec. 14, 2023

Apellis eye drug likely to be rejected in Europe, company says

According to Apellis executives, reviewers in Europe are skeptical of the benefits of its geographic atrophy medicine, Syfovre, and appear poised to recommend against approval at a meeting next month.

By Ben Fidler • Dec. 14, 2023

Bristol Myers pays SystImmune $800M and adds to pharma’s ‘ADC’ deal spree

The lucrative alliance, potentially worth more than $8 billion, continues a run of dealmaking for a class of cancer drugs that’s surged in popularity in recent years. 

By Ben Fidler • Dec. 12, 2023

Tome Biosciences debuts with $213M and a new way to edit the genome

Based on the work of MIT scientists, the well-funded startup is developing ways to insert large sizes of genetic material anywhere in the genome without damaging or breaking DNA.

By Ben Fidler • Dec. 12, 2023

Pricey new gene therapies for sickle cell pose access test

Casgevy, the first CRISPR therapy approved by the FDA, will cost $2.2 million, while Bluebird’s competing genetic medicine is priced at $3.1 million.

By Ned Pagliarulo • Dec. 8, 2023

Spyre Therapeutics raises $180 million to advance anti-inflammatory drugs

The private placement funding will help Spyre develop its drug pipeline, which includes a TL1A-targeting therapy for inflammatory bowel disease.

By Delilah Alvarado • Dec. 8, 2023

CRISPR therapy for sickle cell approved by FDA in gene editing milestone

Developed by Vertex Pharmaceuticals and CRISPR Therapeutics, Casgevy is the first drug based on the Nobel Prize-winning gene editing technology to reach market.

By Ned Pagliarulo • Updated Dec. 8, 2023

Radiopharma startup Artbio raises $90M in sign of field’s momentum

The funding is indicative of investor interest in an area of drug research that involves at least a dozen startups and multiple publicly traded companies.

By Kristin Jensen • Dec. 7, 2023

Biotech venture firms continue ‘recalibration’ in third quarter, Pitchbook report finds

Data collected by Pitchbook suggests venture funding totals are still sliding amid a "strategic shift" by investors towards fewer, but more substantial deals.

By Ben Fidler • Dec. 7, 2023

AbbVie to buy Cerevel in $8.7B bet on brain drugs

The deal hands AbbVie a portfolio of psychiatric medicines that originated within Pfizer, among them a closely watched schizophrenia treatment that’s in late-stage testing.

By Ned Pagliarulo , Jacob Bell • Updated Dec. 6, 2023

Fujifilm to invest $200M in cell therapy production

The funds are meant to boost Fujifilm’s capacity to manufacture cell therapies, a market it expects to grow substantially in the coming years.

By Delilah Alvarado • Dec. 6, 2023

Intuitive’s venture capital arm launches second investment fund

The $150 million fund will back early-stage companies focused on healthcare access, digital health, and precision diagnostics and interventions.

By Susan Kelly • Dec. 5, 2023

BIO names longtime Amicus head John Crowley as new CEO

Crowley, a rare disease advocate who led Amicus for nearly two decades, will take the reins as the industry group seeks to challenge Medicare’s new drug pricing powers.

By Jonathan Gardner • Dec. 5, 2023

CRISPR eyes autoimmune disease in revamp of cell therapy plans

The gene editing biotech is shelving two of its most advanced cancer drugs, and joining a growing group of companies exploring cell-based medicines for inflammatory diseases like lupus.

By Ben Fidler • Dec. 5, 2023