Regenxbio's new manufacturing facility in Rockville, MD (via Regenxbio)

Re­genxbio opens gene ther­a­py man­u­fac­tur­ing fa­cil­i­ty at its Mary­land HQ

Re­genxbio is push­ing deep­er in­to the gene ther­a­py space with the open­ing of a new fa­cil­i­ty at its cam­pus in the Wash­ing­ton D.C., sub­urb of Rockville, MD.

Built in a year, the $65 mil­lion, 132,000-square-foot GMP fa­cil­i­ty will en­able the com­pa­ny to boost its man­u­fac­tur­ing of NAV Tech­nol­o­gy-based ade­no-as­so­ci­at­ed virus, or AAV, vec­tors at scales up to 2,000 liters. The fa­cil­i­ty will al­so im­ple­ment Re­genxbio’s plat­form sus­pen­sion cell cul­ture process.

Ken Mills, Re­genxBio CEO

“We be­lieve our in-house man­u­fac­tur­ing ca­pa­bil­i­ties will en­able us to rapid­ly tran­si­tion pro­duc­tion process­es across the prod­uct life­cy­cle, and ef­fi­cient­ly ad­vance new AAV Ther­a­peu­tics from re­search and ear­ly de­vel­op­ment to clin­i­cal pro­grams to com­mer­cial readi­ness, and in­to the hands of pa­tients who may ben­e­fit from these po­ten­tial one-time ad­min­is­tra­tion ther­a­pies,” said Re­genxbio’s CEO Ken Mills in a state­ment.

The site now in­cludes two in­de­pen­dent bulk drug sub­stance pro­duc­tion suites, a fi­nal drug prod­uct suite and in­te­grat­ed qual­i­ty con­trol labs, and pro­duc­tion at the site be­gan in May.

Mills said in an email to End­points News, that the com­pa­ny re­ceived $800,000 in fi­nanc­ing in­cen­tives from the gov­ern­ment of Mont­gomery Coun­ty and the state of Mary­land for the build­out of their head­quar­ters, which in­cludes the new man­u­fac­tur­ing cen­ter. Aside from an­oth­er es­ti­mat­ed $19.5 mil­lion in ten­ant im­prove­ment al­lowances, the rest of the project was fund­ed di­rect­ly by Re­genxbio. No debt was in­curred re­lat­ed to the build-out.

Last year, the com­pa­ny in­vest­ed more than $100 mil­lion in­to the build­out of its head­quar­ters in Rockville and the com­pa­ny has made over 200 hires in the past two years bring­ing its to­tal to over 400 peo­ple, with more than 100 ded­i­cat­ed to man­u­fac­tur­ing.

How­ev­er, the news of the build-out has done lit­tle to soothe in­vestors, es­pe­cial­ly amidst the cur­rent bear mar­ket woes for biotechs. The com­pa­ny finds its stock price $RGNX down 44% from the be­gin­ning of the year.

The com­pa­ny al­so suf­fered a set­back when No­var­tis told the FDA in Au­gust that Re­genxbio shouldn’t have filed for a PTE  for its AAV9 vec­tor be­cause that ap­pli­ca­tion should have come from No­var­tis.

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.

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Alex­ion puts €65M for­ward to strength­en its po­si­tion on the Emer­ald Isle

Ireland has been on a roll in 2022, with several large pharma companies announcing multimillion-euro projects. Now AstraZeneca’s rare disease outfit Alexion is looking to get in on the action.

Alexion on Friday announced a €65 million ($68.8 million) investment in new and enhanced capabilities across two sites in the country, including at College Park in the Dublin suburb of Blanchardstown and the Monksland Industrial Park in the central Irish town of Athlone, according to the Industrial Development Agency of Ireland.

Members of the G7 from left to right: Prime Minister of Italy Mario Draghi, European Commission President Ursula von der Leyen, President Joe Biden, German Chancellor Olaf Scholz, British Prime Minister Boris Johnson, Canadian Prime Minister Justin Trudeau, Prime Minister of Japan Fumio Kishida, French President Emmanuel Macron and European Council President Charles Michel (AP Photo/Susan Walsh)

Biden and G7 na­tions of­fer funds for vac­cine and med­ical prod­uct man­u­fac­tur­ing project in Sene­gal

Amidst recently broader vaccine manufacturing initiatives from the EU and European companies, the G7 summit in the mountains of Bavaria has brought about some positive news for closing vaccine and medical product manufacturing gaps around the globe.

According to a statement from the White House, the G7 leaders have formally launched the partnership for global infrastructure, PGII. The effort will aim to mobilize hundreds of billions of dollars to deliver infrastructure projects in several sectors including the medical and pharmaceutical manufacturing space.

Fed­er­al judge de­nies Bris­tol My­er­s' at­tempt to avoid Cel­gene share­hold­er law­suit

Some Celgene shareholders aren’t happy with how Bristol Myers Squibb’s takeover went down.

On Friday, a New York federal judge ruled that they have a case against the pharma giant, denying a request to dismiss allegations that it purposely slow-rolled Breyanzi’s approval to avoid paying out $6.4 billion in contingent value rights (CVR).

When Bristol Myers put down $74 billion to scoop up Celgene back in 2019, liso-cel — the CAR-T lymphoma treatment now marketed as Breyanzi — was supposedly one of the centerpieces of the deal. After going back and forth on negotiations for about six months, BMS put $6.4 billion into a CVR agreement that required an FDA approval for Zeposia, Breyanzi and Abecma, each by an established date.

No stranger to gene ther­a­py woes, Astel­las runs in­to an­oth­er safe­ty-re­lat­ed clin­i­cal hold

Astellas Pharma, which has been at the forefront of uncovering the risks associated with gene therapies delivered by adeno-associated viruses, must take another safety alarm head-on.

The FDA has slapped a clinical hold on Astellas’ Phase I/II trial of a gene therapy candidate for late-onset Pompe disease, after investigators flagged a serious case of peripheral sensory neuropathy.

It marks the latest in a streak of setbacks Astellas has encountered since making a splashy entry into the gene therapy space with its $3 billion buyout of Audentes. But the lead program, AT132 for the treatment of X-linked myotubular myopathy (XLMTM), had to be halted more than once after a total of four patients died in the trial — and the scientific community still doesn’t have all the answers of what caused the deaths.

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Linda Marbán, Capricor CEO

Capri­cor hits pri­ma­ry end­point in Duchenne ex­ten­sion study, ready­ing its FDA pitch

It has been a very bumpy ride for Capricor Therapeutics over the past several years, including a halted J&J partnership and venture into penny stock status. However, the latest news from the San Diego-based biotech shows that its experimental Duchenne drug may be a durable, long-term treatment.

The company posted the one-year results from its open-label extension study for their candidate dubbed CAP-1002. The extension from its Phase II study, named HOPE-2, was conducted in boys and young men with later-stage Duchenne muscular dystrophy who paused and then resumed treatment after a year. Researchers measured patients in the one-year follow-up against the original study’s treatment arm effect and in two subsets of the original placebo group.

AstraZeneca's new Evusheld direct to consumer campaign aims to reach more immunocompromised patients.

As­traZeneca de­buts first con­sumer cam­paign for its Covid-19 pro­phy­lac­tic Evusheld — and a first for EUA drugs

AstraZeneca’s first consumer ad for Evusheld is also a first for drugs that have been granted emergency use authorizations during the pandemic.

The first DTC ad for a medicine under emergency approval, the Evusheld campaign launching this week aims to raise awareness among immunocompromised patients — and spur more use.

Evusheld nabbed emergency authorization in December, however, despite millions of immunocompromised people looking for a solution and now more widespread availability of the drug.

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Am­gen takes next step with its Chi­na am­bi­tions, out-li­cens­ing drugs to Fo­s­un Phar­ma

In a bid to increase its market share in China, Amgen has agreed to a partnership with a Shanghai biotech — a collaboration and out-licensing agreement for two of its drugs.

Amgen and Fosun Pharma announced a deal Monday in a bid to increase Amgen’s presence in the country. The stated goal so far is to commercialize Amgen’s blockbuster psoriasis drug Otezla alongside Parsabiv, a drug for secondary hyperparathyroidism in adults with chronic kidney disease and on a specific type of dialysis.

As court case looms, Bris­tol My­ers touts la­bel ex­pan­sion for Breyanzi

As Bristol Myers Squibb braces for a court battle over a costly delay — at least for Celgene shareholders — for its CAR-T lymphoma treatment Breyanzi, the pharma giant is touting a label expansion in the second-line setting.

Breyanzi, also known as liso-cel, snagged a win on Friday in adults with large B-cell lymphoma (LBCL) who: don’t respond to chemotherapy, or relapse within 12 months; don’t respond or relapse after 12 months; or are not eligible for hematopoietic stem cell transplant after chemo due to their age or comorbidities.