Feng Zhang (Susan Walsh/AP Images)

Broad In­sti­tute lands ma­jor vic­to­ry in CRISPR patent fight over UC Berke­ley, No­bel win­ners

Jen­nifer Doud­na

Em­manuelle Char­p­en­tier and Jen­nifer Doud­na have their No­bel prizes in chem­istry for their CRISPR-Cas9 DNA scis­sors, but the US Patent and Trade­mark Of­fice made clear late Mon­day that Har­vard-MIT’s Broad In­sti­tute and su­per­star re­searcher Feng Zhang con­trol the patents in­volv­ing claims to CRISPR-Cas9 sys­tems for use in eu­kary­ot­ic (have a nu­cle­us) cells.

The de­ci­sion was an­oth­er huge le­gal blow to the No­bel prize win­ners, as well as the Uni­ver­si­ty of Cal­i­for­nia and Uni­ver­si­ty of Vi­en­na, and com­pa­nies like In­tel­lia Ther­a­peu­tics and Char­p­en­tier-found­ed CRISPR Ther­a­peu­tics, which do not have li­cens­es with the Broad In­sti­tute. In­tel­lia, which al­so re­leased some ear­ly CRISPR da­ta on Mon­day, saw its stock price fall by about 9% af­ter hours on Mon­day.

Oth­er com­pa­nies like Ed­i­tas Med­i­cine, which has li­censed IP from the Broad, cel­e­brat­ed the de­ci­sion.

Em­manuelle Char­p­en­tier

“The de­ci­sion reaf­firms the strength of our foun­da­tion­al in­tel­lec­tu­al prop­er­ty as we con­tin­ue our work to de­vel­op life-chang­ing med­i­cines for peo­ple liv­ing with se­ri­ous dis­eases,” Ed­i­tas CEO James Mullen said in a state­ment.

The de­ci­sion Mon­day al­so reaf­firms sev­er­al past court de­ci­sions. Back in 2018, the US Court of Ap­peals for the Fed­er­al Cir­cuit de­cid­ed that the piv­otal IP be­longed to the Broad, up­hold­ing a 2017 de­ci­sion by the USP­TO.

The patent in ques­tion, orig­i­nal­ly grant­ed in 2014, be­gan with work at the Broad and MIT in ear­ly 2011, and cul­mi­nat­ed in a pub­li­ca­tion in Sci­ence in Jan. 2013.

Jim Mullen

“This marked the world’s first en­gi­neer­ing of CRISPR-Cas9 to be de­liv­ered and used to achieve mam­malian genome edit­ing. Zhang was the first to file a patent ap­pli­ca­tion that de­scribed and en­abled such a method,” the Broad ex­plains.

De­spite the patent, and le­gal tus­sle, Zhang, Doud­na and Char­p­en­tier have sought to make their in­ven­tions more wide­ly ac­ces­si­ble. Since 2013, the in­sti­tute says that Zhang’s lab has open­ly shared CRISPR reagents and tools with more than 3,000 in­sti­tu­tions in 75 coun­tries through the non­prof­it Ad­dgene.

Uni­ver­si­ty of Illi­nois law pro­fes­sor Ja­cob Sherkow, who has been fol­low­ing the le­gal ter­rain around CRISPR-Cas9 close­ly over the years, shared his thoughts in a long thread on the re­sult yes­ter­day, adding, “I don’t think an ap­peal is go­ing any­where.”

Jean-Paul Clozel, Idorsia CEO (Patrick Straub/Keystone via AP Images)

Idor­si­a's brain bleed drug flunks PhI­II tri­al, a decade af­ter pre­vi­ous flop

Idorsia’s long journey with clazosentan came to an abrupt “unexpected result” Monday morning with a Phase III flop.

The Swiss biopharma said the drug did not meet the main goal of the late-stage REACT study, conducted in the US, Canada and Europe since early 2019.

The 409-patient trial tested the intravenous drug’s ability to prevent complications due to delayed cerebral ischemia following aneurysmal subarachnoid hemorrhage (aSAH), in which blood vessels in the brain narrow and blood accumulates around the brain’s surface, which then dials up the pressure on the brain.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 158,600+ biopharma pros reading Endpoints daily — and it's free.

Kenji Yasukawa, Astellas CEO (Photographer: Akio Kon/Bloomberg via Getty Images)

Astel­las taps chief strat­e­gy of­fi­cer as next CEO to 'go on the ag­gres­sive'

Five years into its big R&D revamp, Astellas says it’s time for a changing of the guard.

Kenji Yasukawa, who took over as president and CEO in 2018, will step down to become chairman of the board in April, making room for Naoki Okamura to take over. Okamura joined the company in 1986 and has served in a variety of finance, business and strategy roles, including most recently as chief strategy officer.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 158,600+ biopharma pros reading Endpoints daily — and it's free.

Clin­i­cal tri­al di­ver­si­ty da­ta show mis­match be­tween en­roll­ment and dis­ease preva­lence, GSK says

A lack of diversity in clinical trials has persisted despite decades of initiatives to try to turn the tide.

In a recent review of 17 years of clinical trials, drugmaker GSK found that there were some mismatches between the demographics of its US-based trials and how prevalent diseases were in those populations.

The results, the company says, will help GSK and others design studies that better represent epidemiological rates within races and ethnicities.

The Big Phar­ma dis­card pile; Lay­offs all around while some biotechs bid farewell; New Roche CEO as­sem­bles top team; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

With earnings seasons in full swing, we’ve listened in on all the calls so you don’t have to. But news is popping up from all corners, so make sure you check out our other updates, too.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 158,600+ biopharma pros reading Endpoints daily — and it's free.

Sen. Ron Wyden (D-OR) (Francis Chung/E&E News/Politico via AP Images)

In­fla­tion re­bates in­com­ing: Wyden calls on CMS to move quick­ly as No­var­tis CEO pledges re­ver­sal

Senate Finance Chair Ron Wyden (D-OR) this week sent a letter to the head of the Centers for Medicare & Medicaid Services seeking an update on how and when new inflation-linked rebates will take effect for drugs that see major price spikes.

The newly signed Inflation Reduction Act requires manufacturers to pay a rebate to Medicare when they increase drug prices faster than the rate of inflation.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 158,600+ biopharma pros reading Endpoints daily — and it's free.

Lina Khan, FTC chair (Graeme Jennings/Pool via AP)

FTC makes an ex­am­ple of GoodRx, bans dis­counter from shar­ing pri­vate health da­ta with ad­ver­tis­ers

Prescription drug discount provider GoodRx will no longer be allowed to share its users’ sensitive health data with advertisers after the Federal Trade Commission charged the online coupon provider with failing to notify consumers of such disclosures to Facebook, Google, and other companies.

GoodRx agreed to pay a $1.5 million civil penalty for violating the FTC’s Health Breach Notification Rule after the FTC said it repeatedly violated a 2017 promise to not share sensitive personal health information. The FTC alleged that the company shared users’ prescription medications and personal health conditions with third party advertisers and platforms like Facebook, Google, Criteo, Branch and Twilio.

Uğur Şahin, BioNTech CEO (Andreas Arnold/picture-alliance/dpa/AP Images)

BioN­Tech opens new plas­mid DNA man­u­fac­tur­ing fa­cil­i­ty in Ger­many

German mRNA player BioNTech opened the doors to a new manufacturing facility on Thursday, this one just about 75 miles north of its headquarters in Mainz, Germany.

BioNTech announced on Thursday that it has completed the construction of its first plasmid DNA manufacturing facility in Marburg, Germany. The facility will produce materials for mRNA-based vaccines and therapies along with cell therapies.

David Kirn, 4D Molecular Therapeutics CEO (via website)

FDA places hold on 4D Mol­e­c­u­lar’s Fab­ry gene ther­a­py

4D Molecular Therapeutics quietly tucked an FDA clinical hold on its Fabry gene therapy into an SEC filing.

Meanwhile, the biotech issued a press release the same day after the closing bell on Thursday touting an IND for another asset, in diabetic macular edema.

The California biotech had paused enrollment of patients in its two trials of the Fabry gene therapy (4D-310) last month after three patients experienced kidney issues, all of which were resolved within four weeks. At the time, 4DMT said it would wait until the second half of this year to look at 12-month clinical data on six patients in the Phase I/II trials, one in the US and one in Taiwan and Australia.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 158,600+ biopharma pros reading Endpoints daily — and it's free.

Trodelvy notch­es a win in most com­mon form of breast can­cer

Following a promise last year to go “big and fast in breast cancer,” Gilead has secured a win for Trodelvy in the most common form.

The drug was approved to treat HR-positive, HER2-negative breast cancer patients who’ve already received endocrine-based therapy and at least two other systemic therapies for metastatic cancer, Gilead announced on Friday.

Trodelvy won its first indication in metastatic triple-negative breast cancer back in 2020, and has since added urothelial cancer to the list. HR-positive HER2-negative breast cancer accounts for roughly 70% of new breast cancer cases worldwide per year, according to senior VP of oncology clinical development Bill Grossman, and many patients develop resistance to endocrine-based therapies or worsen on chemotherapy.