Armed with new gene therapy tools, a biotech startup promises a better way to target muscle diseases

Two-year-old Kate Therapeutics is launching publicly with $51 million and a licensing deal with Astellas Pharma for a neuromuscular disease gene therapy.

By Ned Pagliarulo • June 8, 2023

Ahead of key meeting, FDA appears open to full approval of Alzheimer’s drug Leqembi

Agency advisers are meeting Friday to discuss whether recent trial data confirm Leqembi’s benefit. A vote is expected in the late afternoon. 

By Jacob Bell • June 7, 2023

Explore the Trendline➔

The expanding world of RNA therapies

Medicines from Alnylam, Ionis, Moderna and others have proven the power of RNA in medicine. A growing field of startups is advancing alongside them.

By BioPharma Dive staff

With new startup Bitterroot, Forty Seven founder takes aim at heart disease

Co-founded by Stanford University professor Irv Weissman, the company debuted with $145 million and plans to use CD47 antibodies, best known as potential cancer immunotherapies, to reduce the risk of heart attacks. 

By Ben Fidler • June 7, 2023

Using twin viruses, startup AAVantgarde aims to extend gene therapy’s reach

The biotech has raised about $65 million to test two ways to deliver larger genes into the body, which could help gene therapy treat more diseases.

By Ben Fidler • June 6, 2023

FDA sets advisory meeting date for Brainstorm’s ALS cell therapy

Having taken the rare step of filing for approval over protest, Brainstorm will get another chance to make a case for its drug NurOwn during a Sept. 27 meeting of cell, tissue and gene therapy experts.

By Jacob Bell • June 6, 2023

Tasked with steadying BIO, Rachel King faces biotech’s many challenges

As the lobbying group holds its annual meeting in Boston, BIO’s interim leader is trying to focus attention back on the industry’s most pressing priorities, like pushing back on the IRA. 

By Alexandra Pecci • June 5, 2023

An unconventional drug startup lures Vertex founder Josh Boger back to biotech

The pioneering executive has rejoined small startup Alkeus Pharmaceuticals to help launch a Stargardt disease treatment he’s long viewed as a “perfect” drug.

By Ben Fidler • June 5, 2023

Startup Eikon, led by Merck vets, buys drugs from three biotechs and banks another $106M

The California biotech, led by Roger Perlmutter and other former Merck executives, has now raised nearly $775 million since its launch two years ago.

By Gwendolyn Wu • June 1, 2023

Biohaven sends latest drug to FDA, despite past trial setback

The biotech, best known for its success developing migraine medicines, will seek approval of a treatment that failed a Phase 3 trial but showed signs of a positive effect.

By Delilah Alvarado • May 31, 2023

Rain Oncology to cut workforce by 65% after study failure

The company, which went public in 2021 to develop a cancer drug licensed from Daiichi Sankyo, may try to acquire a different tumor-targeting medicine to grow its pipeline.

By Ben Fidler • May 30, 2023

Atea rejects takeover bid from Concentra

The COVID-19 drug developer said the offer from the firm that recently took over Jounce Therapeutics “fundamentally undervalues the company.”

By Gwendolyn Wu • May 30, 2023

Amylyx’s ALS drug could face rejection in Europe

The biotech said a committee advising the European Medicines Agency is “trending” toward issuing a negative opinion of its drug Relyvrio, which was approved in the U.S. last year. 

By Delilah Alvarado • May 30, 2023

Slump in medtech VC activity continues as inflation keeps investors on the sidelines

Investors struck deals worth $1.8 billion over the first three months of the year, down from $3 billion in early 2022.

By Nick Paul Taylor • May 26, 2023

Serial biotech founder Tim Springer on investing in startups and democratizing antibody research

Springer, who became a billionaire after investing in Moderna, highlighted gene therapy and machine learning as two fields he’s optimistic will shape biotech’s future. 

By Gwendolyn Wu • May 26, 2023

Illumina chair loses to Icahn pick as proxy battle ends

The decision to replace board Chair John Thompson could change the company’s course as it decides whether to divest liquid biopsy company Grail.

By Elise Reuter • Updated May 25, 2023

BenevolentAI CFO resigns as company lays off staff, restructures

The biotech, one of several AI drug discovery specialists to go public in recent years, is cutting up to 180 jobs and reorganizing its pipeline to conserve cash.

By Delilah Alvarado • May 25, 2023

Digital health funding holds steady in Q1 after year of decline

The sector defied trends seen in the broader venture capital economy, where funding has tightened. But investment is still at years-low levels.

By Emily Olsen • May 25, 2023

Illumina, Icahn prepare for proxy showdown

Can Carl Icahn bend Illumina to his will? Shareholders will vote Thursday on removing the company’s CEO and chairman from the board and replacing them with nominees from the activist investor.

By Elise Reuter • May 25, 2023

PTC lays off staff, fires CFO as neuromuscular drug study fails

The biotech is giving up on early-stage gene therapy research as part of a restructuring meant to cut costs and redirect resources toward therapies most likely to succeed.

By Kristin Jensen • May 24, 2023

FDA delays decision on Duchenne gene therapy, considers narrower approval

The agency will extend its review of Sarepta’s treatment by one month as it weighs limiting an initial OK to children with Duchenne aged 4 to 5 years old.

By Ben Fidler • Updated May 24, 2023

Concentra bids on Atea, seeking to acquire another struggling biotech

However, Atea’s board said Tuesday they rejected the offer from Concentra, which was recently involved in the takeover of Jounce Therapeutics.

By Gwendolyn Wu • Updated May 30, 2023

ReNAgade debuts with $300M and an ‘all-in-one’ system for delivering RNA drugs

Staffed by former Alnylam and Moderna employees, the startup is developing technologies that could be useful in a variety of RNA drugmaking methods.

By Gwendolyn Wu • May 23, 2023

Novartis buys rare disease gene therapy from Avrobio

The pharma will pay nearly $90 million to acquire Avrobio’s treatment for cystinosis, an inherited condition caused by the toxic buildup of an amino acid.

By Ned Pagliarulo • May 22, 2023

How the digital front door benefits clinical trial patients, clinicians and sponsors

A digital front door in clinical trials is a triple win that improves patient and clinician satisfaction, data quality and costs.

By Ben Baumann, OpenClinica Co-Founder and COO • May 22, 2023

Moderna reports ‘encouraging’ early data for one of its rare disease medicines

Now known for its vaccine research, the biotech said its clinical trial is the first to report results of a messenger RNA therapeutic designed for intracellular protein replacement.

By Jacob Bell • May 19, 2023