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In this 2021 photo, Lexi Worth of Bloomington, Illinois, held her son Axel Riordan, who suffered with the rare genetic disease metachromatic leukodystrophy. Orchard Therapeutics, whose US headquarters is located in Boston, recently won FDA approval for a therapy for the disease. The one-time treatment lists at $4.25 million — the most expensive ever approved.

Biotech with links to Boston sets record price for new gene therapy for rare childhood disease

The gene therapy, which won the approval of the Food and Drug Administration on Monday, is the most expensive drug ever approved, clocking in at $4.25 million for a one-time treatment.

‘Kendall Square Jr.’ Another big biotech firm sets up shop in the Seaport.
About 800 workers for the biotechnology firm have moved into the 630,000 square-foot-building, with the remaining 300 expected to arrive by early next year.

The Boston area built a ton of lab space. Now many of those buildings are opening empty.
The region's supply of life science real estate exploded just as the once-booming industry hit a slow patch. Now what?

‘Nothing else that has had this effect’: New treatment shrinks brain tumors in small clinical trial at Mass. General
The treatment involved a new form of CAR-T cell therapy, which extracts and reprograms a patient’s immune cells to help fight the disease.

In setback for ALS patients, Cambridge biotech’s drug fails clinical trial, could be withdrawn from market
The drug, called Relyvrio, was approved in September 2022 even though an advisory panel to the FDA cited weak scientific evidence in its recommendation that the agency reject it.

Alnylam’s experimental drug for high blood pressure yields encouraging results
Alnylam has mostly focused on drugs that treat rare diseases. If the high blood pressure medicine wins regulatory approval, it would represent a significant pivot for the Cambridge biotech.

Low-cost versions of Humira, the nation’s top-selling drug, are off to a sluggish start
The failure of Humira’s competitors to gain much traction — so far, at least — helps illustrate the market grip maintained by brand-name drug makers like AbbVie, despite the $84,000-a-year price tag of its wildly successful anti-inflammatory drug.

‘It’s a nightmare’: One of the most common children’s asthma meds is no longer available, leaving families scrambling
Senator Elizabeth Warren on Friday accused the drug's manufacturer, GlaxoSmithKline, of “price gouging.”

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More Life Sciences Headlines

A scientist working at SQZ Biotechnologies in Watertown's Arsenal Yards.

Healey’s life sciences plan: Another $1 billion investment, with some new areas of focus

Lawmakers are so far noncommittal on funding the proposal, which will be part of a broader economic development bill the Healey administration is expected to file soon. 9:54 a.m.

Moderna stock rises as COVID shot gains market share on Pfizer

Shares of the Cambridge-based biotech were up in early trading Thursday after the company reported fourth-quarter revenue that beat analysts’ expectations by gaining COVID vaccine market share on its main rival. 9:17 a.m.

Tom DiLenge (left), of Flagship Pioneering in Cambridge, talking with US Representative Raja Krishnamoorthi of Illinois at a biotech fair hosted by the Harvard Kennedy School in Cambridge where members of the House Select Committee on the Chinese Communist Party talked to local industry leaders about the complexities of doing business with China.

US lawmakers, visiting Cambridge, warn of a rising Chinese threat to US biotech

A bipartisan group of lawmakers visited Mass. this week to call for strengthening US life sciences companies, protecting their technologies, and safeguarding Americans’ genetic data. 12:38 p.m.

Astrida Schaeffer received an infusion of Leqembi at Brigham and Women's Hospital last summer. Seven months after the drug received FDA approval, only about 2,000 patients are taking the medicine nationwide, the drug's maker, Biogen, said Tuesday.

Biogen Alzheimer’s drug launch off to slow start

Only about 2,000 patients are taking the medicine nationwide, but Biogen’s chief executive, Christopher A. Viehbacher, said he had expected the slow uptake and sees considerable demand for the medication. 8:36 a.m.

Jonathan Gootenberg, left, and Omar Abudayyeh, cofounders of Tome Biosciences, a Watertown startup that reengineers genes and cells to cure diseases.

A Jewish American and a Palestinian American are tackling science’s toughest challenges

Omar Abudayyeh and Jonathan Gootenberg have co-published 10 scientific papers, launched several biotech companies, and jointly run a Harvard lab. 10:52 a.m.

Elemind Technologies said it has closed on a seed round of $12 million in funding from a consortium that includes Village Global, an early-stage venture fund backed by Jeff Bezos and other tech billionaires.

This Cambridge startup, bootstrapped by billionaires, is developing ‘neuro-tech’ wearables

The company is designing products based on “electric medicine,” an approach that redirects brain waves through non-invasive stimulation — using sound, light, touch, and electrical pulses. 3:56 p.m.

An artist's rendering of King Street Properties' planned Pathway Triangle biomanufacturing campus in Morrisville, N.C.

A looming threat to Greater Boston’s biotech dominance, 700 miles away

Boston-based firms that build biomanufacturing space see big opportunity in Raleigh-Durham — posing a threat that a key pillar of our region’s economy could grow faster elsewhere. 8:33 a.m.

Biogen is giving up its ownership of Aduhelm, the Alzheimer’s disease treatment whose 2021 approval led to scrutiny and outrage.

Biogen plans to shut down its controversial Alzheimer’s drug Aduhelm

The controversial approval of the Alzheimer’s disease treatment in 2021 led to scrutiny and outrage. 9:31 a.m.