Sang­amo claims a ma­jor mile­stone in gene edit­ing, treat­ing first pa­tient with its zinc fin­ger nu­cle­ase tech

While the lead­ing CRISPR biotechs have been fo­cused on a long, slow windup to their first hu­man stud­ies, one of the orig­i­nal play­ers in the gene edit­ing field has scored boast­ing rights for a first-in-hu­man ex­per­i­ment.

Sang­amo Ther­a­peu­tics $SG­MO has treat­ed its first pa­tient in a Phase I/II tri­al for a rare dis­ease called Hunter syn­drome, or mu­copolysac­cha­ri­do­sis type II — MPS II. Physi­cians tar­get­ed the liv­er with SB-913, which us­es Sang­amo’s zinc fin­ger nu­cle­ase tech de­liv­ered via AAV cells with a cor­rec­tive gene. The ZFNs snip the tar­get­ed liv­er cells to in­sert the cor­rec­tive gene at a spec­i­fied point.

Sandy Macrae, CEO

By re­pair­ing the cells Sang­amo says they should be good to go in pro­duc­ing an en­zyme called iduronate-2-sul­fa­tase, which is used to rid the cells of car­bo­hy­drates that ac­cu­mu­late in a tox­ic load, the hall­mark of MPS II.

“Even with reg­u­lar in­fu­sions of ERT, which has marked­ly im­proved func­tion­al health out­comes, pa­tients en­dure pro­gres­sive dam­age to heart, bones and lungs. Many pa­tients with MPS II die of air­way ob­struc­tion, up­per res­pi­ra­to­ry in­fec­tion or heart fail­ure be­fore they reach the age of 20,” said Paul Har­matz, a pe­di­atric gas­troen­terol­o­gist and a prin­ci­pal in­ves­ti­ga­tor for the CHAM­PI­ONS study.

Gene edit­ing is an emerg­ing field in the biotech world, and the top play­ers here — in­clud­ing Ed­i­tas, In­tel­lia and CRISPR Ther­a­peu­tics — have been scrap­ping over con­trol of the CRISPR/Cas IP, even as re­searchers con­tin­ue to work on new and bet­ter ways to go about this kind of DNA surgery. Sang­amo, mean­while, has la­bored in this field for more than 20 years with its ZFN tech, while TAL­EN is the fo­cus at Cel­lec­tis.

The AP iden­ti­fied Sang­amo’s pa­tient 1 as 44-year-old Bri­an Madeux.

“It’s kind of hum­bling” to be the first to test this, Madeux told Reuters. “I’m will­ing to take that risk. Hope­ful­ly it will help me and oth­er peo­ple.”

Scoop: Boehringer qui­et­ly shut­ters a PhII for one of its top drugs — now un­der re­view

Boehringer Ingelheim has quietly shut down a small Phase II study for one of its lead drugs.

The private pharma player confirmed to Endpoints News that it had shuttered a study testing spesolimab as a therapy for Crohn’s patients suffering from bowel obstructions.

A spokesperson for the company tells Endpoints:

Taking into consideration the current therapeutic landscape and ongoing clinical development programs, Boehringer Ingelheim decided to discontinue our program in Crohn’s disease. It is important to note that this decision is not based on any safety findings in the clinical trials.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Lina Gugucheva, NewAmsterdam Pharma CBO

Phar­ma group bets up to $1B-plus on the PhI­II res­ur­rec­tion of a once dead-and-buried LDL drug

Close to 5 years after then-Amgen R&D chief Sean Harper tamped the last spade of dirt on the last broadly focused CETP cholesterol drug — burying their $300 million upfront and the few remaining hopes for the class with it — the therapy has been fully resurrected. And today, the NewAmsterdam Pharma crew that did the Lazarus treatment on obicetrapib is taking another big step on the comeback trail with a €1 billion-plus regional licensing deal, complete with close to $150 million in upfront cash.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 144,400+ biopharma pros reading Endpoints daily — and it's free.

How pre­pared is bio­phar­ma for the cy­ber dooms­day?

One of the largest cyberattacks in history happened on a Friday, Eric Perakslis distinctly remembers.

Perakslis, who was head of Takeda’s R&D Data Sciences Institute and visiting faculty at Harvard Medical School at the time, had spent that morning completing a review on cybersecurity for the British Medical Journal. Moments after he turned it in, he heard back from the editor: “Have you heard what’s going on right now?”

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Scoop: Roche scraps one of two schiz­o­phre­nia PhII tri­als af­ter fail­ing the pri­ma­ry end­point

Roche has terminated one of two Phase II trials testing its drug ralmitaront in patients with schizophrenia, the Big Pharma confirmed to Endpoints News.

The study was terminated last month, according to a June 22 update to the registry on clinicaltrials.gov. Begun in September 2020, the trial was looking at ralmitaront in patients with acute schizophrenia. The trial enrolled 286 patients out of an originally planned 308.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Matt Gline, Roivant CEO (John Sciulli/Getty Images for GLG)

Roivant chops sick­le cell gene ther­a­py, der­ma­tol­ogy drugs to fo­cus on 'high­er val­ue pro­ject­s'

Roivant is sweeping a suite of drugs, including a gene therapy for sickle cell disease already in the clinic, out of its pipeline.

Six programs from four of its “vants” are being wound down as part of “a company-wide cost optimization and pipeline reprioritization initiative to reduce our expected operating expenses and prioritize our capital resources.”

When reached by Endpoints News, a spokesperson said, “We don’t anticipate a material reduction in headcount but we will likely reassign some folks to higher value projects as part of winding down specific programs.”

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 144,400+ biopharma pros reading Endpoints daily — and it's free.

Laurence Reid, Decibel CEO

Still in pre­clin­i­cal test­ing for ear gene ther­a­pies, Deci­bel touts small snap­shot of chemo-in­duced hear­ing loss drug

Though Decibel Therapeutics has largely pivoted toward gene therapies for the inner ear, its lead clinical candidate simply aims to protect cancer patients from chemotherapy-induced hearing loss. On Tuesday, the biotech presented its first efficacy data for the program, and execs like what they see.

Decibel reported interim results from a Phase Ib study showing the experimental drug, dubbed DB-020, largely protected a small group of patients from losing their hearing. Researchers used a particularly unique study design, administering the compound in one of each patients’ ears before they received cisplatin chemotherapy and placebo in the other.

Years af­ter link­ing arms with Bris­tol My­ers and both Mer­cks, Sutro finds its lat­est part­ner in Tokyo

Astellas and Sutro Biopharma are linking arms on a new field of antibody-drug conjugates that they hope will improve upon existing cancer immunotherapies.

The Tokyo pharma will dole out $90 million in cash for the collaboration, the companies said Monday afternoon. That upfront payment will extend the South San Francisco biotech’s runway from late 2023 into the first half of 2024, Cowen analysts noted.

Pearl Huang, Dunad Therapeutics CEO (Ken Richardson, PR Newswire)

Long­time biotech leader Pearl Huang takes the reins as CEO of No­var­tis-backed up­start

It has only been a few months since Pearl Huang exited the top seat at Cygnal Therapeutics, but now she’s back at the helm of another biotech.

After taking a few months off — passing an exam in that time to get her captain’s license from the US Coast Guard — she’s been named CEO of Dunad Therapeutics, a biotech focused on developing a small molecule covalent therapies that was founded in 2020. Huang told Endpoints News that two factors attracted her to going back to the c-suite: the company’s technology and its co-founders.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 144,400+ biopharma pros reading Endpoints daily — and it's free.

Deborah Dunsire, Lundbeck CEO

Af­ter a 5-year re­peat PhI­II so­journ, Lund­beck and Ot­su­ka say they're fi­nal­ly ready to pur­sue OK to use Rex­ul­ti against Alzheimer's ag­i­ta­tion

Five years after Lundbeck and their longtime collaborators at Otsuka turned up a mixed set of Phase III data for Rexulti as a treatment for Alzheimer’s dementia-related agitation, they’ve come through with a new pivotal trial success they believe will finally put them on the road to an approval at the FDA. And if they’re right, some analysts believe they’re a short step away from adding more than $500 million in annual sales for the drug, already approved in depression and schizophrenia.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 144,400+ biopharma pros reading Endpoints daily — and it's free.