J&J snags rights to a gene ther­a­py port­fo­lio from lit­tle MeiraGTx, a spin­off from Sam Wak­sal’s Kad­mon

Af­ter get­ting a quick les­son on lit­tle MeiraGTx’s gene ther­a­py ef­forts — in­clud­ing their home made on/off switch — J&J is go­ing all in. And like oth­er deals it’s made re­cent­ly, J&J’s re­search or­ga­ni­za­tion un­der Math­ai Mam­men is demon­strat­ing its en­thu­si­asm for small biotechs that are short on his­to­ry and long on promise.

The phar­ma gi­ant fol­lowed up on the ini­tial re­search al­liance they signed last fall with the biotech and snapped up com­mer­cial rights to a slate of clin­i­cal-stage, once-and-done de­vel­op­ment pro­grams aimed at eye dis­eases. J&J will pay $100 mil­lion in a cash up­front for the deal and is putting an­oth­er $340 mil­lion on the ta­ble in mile­stones.

Alexan­dria Forbes

J&J is al­so on the hook now for de­vel­op­ment costs for the port­fo­lio, which in­cludes can­di­dates for in­her­it­ed reti­nal dis­eases: achro­matop­sia caused by mu­ta­tions in ei­ther CNGB3 or CN­GA3 and X-linked re­tini­tis pig­men­tosa. Tied in is a pact on col­lab­o­rat­ing on AAV man­u­fac­tur­ing tech­nol­o­gy, which they will work on to­geth­er.

J&J is ty­ing it­self up to a New York-based com­pa­ny that spun out of Kad­mon, Sam Wak­sal’s come­back biotech af­ter his prison sen­tence for in­sid­er trad­ing — a case that al­so took celebri­ty home­mak­er Martha Stew­art down with him. Wak­sal bowed out just as Kad­mon went pub­lic, as he was barred from be­ing an of­fi­cer in a pub­lic com­pa­ny again.

J&J cre­at­ed an ex­ten­sive net­work of deal­mak­ing of­fices around the world sev­er­al years ago, and it’s been pay­ing some top prices to land new clin­i­cal-stage drugs to bring in­to the pipeline. That strat­e­gy was on dis­play when J&J snagged the BC­MA CAR-T at Shang­hai’s Leg­end, with plans to go up against blue­bird for the first-in-class ti­tle.

“Our gene reg­u­la­tion plat­form, based on RNA struc­ture, was in­vent­ed and de­vel­oped en­tire­ly in-house at MeiraGTx and is a re­flec­tion of our com­mit­ment to ground-break­ing sci­ence,” said MeiraGTx CEO Alexan­dria Forbes, a sci­en­tist who took a lat­er spin at a hedge fund be­fore helm­ing her first biotech.

Op­ti­miz­ing Cell and Gene Ther­a­py De­vel­op­ment and Pro­duc­tion: How Tech­nol­o­gy Providers Like Corn­ing Life Sci­ences are Spurring In­no­va­tion

Remarkable advances in cell and gene therapy over the last decade offer unprecedented therapeutic promise and bring new hope for many patients facing diseases once thought incurable. However, for cell and gene therapies to reach their full potential, researchers, manufacturers, life science companies, and academics will need to work together to solve the significant challenges facing the industry.

Pfiz­er, Sarep­ta and two oth­ers sug­gest Duchenne drug safe­ty is­sues tied to "class ef­fect"

Since the first experimental Duchenne gene therapy programs came about, the space has proven rife with safety issues and patient deaths in clinical trials. Pfizer and three biotechs now think they’ve found a reason why.

The four companies suggested there may be a “class effect” causing the adverse events in Duchenne gene therapies, they wrote in a new study. They specifically highlighted how side effects in five patients across three trials, who all showed muscle weakness with cardiac involvement, were “strikingly similar.”

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Pre­sent­ing a live End­points News event: Man­ag­ing a biotech in tur­bu­lent times

Biotech is one of the smartest, best educated industries on the planet. PhDs abound. We’ve had a long enough track record to see a new generation of savvy, experienced execs coming together to run startups.

And in these times, they are being tested as never before.

Biotech is going through quite a rough patch right now. For 2 years, practically anyone with a decent resume and some half-baked ideas on biotech could start a company and get it funded. The pandemic made it easy in many ways to pull off an IPO, with traditional road shows shut down in exchange for a series of quick Zoom meetings. Generalist investors flocked as the numbers raised soared into the stratosphere.

De­spite fed­er­al ef­forts to di­ver­si­fy clin­i­cal tri­als, progress re­mains 'stag­nan­t' — re­port

While calls to diversify clinical trials have grown louder in recent years — gaining support from federal agencies such as the FDA and NIH — progress has largely stalled, according to a new report from the National Academies of Sciences, Engineering and Medicine.

Swaths of patients in racial and ethnic minority groups, as well as LGBTQIA+, pregnant and older adult populations continue to be left out of clinical trials. While some advances have been made in the last 30 years — women now account for roughly half of clinical trial participants — growth in other areas remains stagnant, according to the report, which was mandated by Congress and sponsored by the NIH.

Paul Chaplin, Bavarian Nordic president and CEO

Bavar­i­an Nordic se­cures BAR­DA con­tract for small­pox vac­cine

It seems that smallpox vaccination production is weighing on the mind of the US government. And manufacturer Bavarian Nordic is the latest company to benefit.

Just a few days after Emergent, a company that has made government contracts its lifeblood, acquired the exclusive rights to Tembexa from Chimerix, with a $225 million cash payment and an expected BARDA contract, the agency has offered a contract for smallpox vaccine production.

Lina Khan, FTC chair (Saul Loeb/Pool via AP)

New FTC com­mis­sion­er could turn the tide for an in­ves­ti­ga­tion in­to PBMs

The Senate last week voted along party lines, 51-50, with Vice President Kamala Harris casting the tie-breaker, to make President Biden appointee Alvaro Bedoya the deciding vote on a split 2-2 Federal Trade Commission.

The addition of Bedoya to the FTC could not only spell more trouble for biopharma M&A activity, as he may align with his Democrat partners to break the FTC ties, but it may also mean that FTC Chair Lina Khan has what she needs to move forward on a study around the pharma middlemen known as pharmacy benefit managers.

Patty Murray (D-WA) (Graeme Sloan/Sipa USA)(Sipa via AP Images)

Sen­ate user fee reau­tho­riza­tion bill omits ac­cel­er­at­ed ap­proval re­forms, shows wide gaps with House ver­sion

The Senate health committee on Tuesday released its first version of the bill to reauthorize all the different FDA user fees. But unlike the House version, there are only a few controversial items in the Senate’s version, which does not address either accelerated approval reforms or clinical trial diversity (as the House did).

While it’s still relatively early in the process of finalizing this legislation (the ultimate statutory deadline is the end of September), the House and Senate, at least initially, appear to be starting off in different corners on what should be included.

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Warren Buffett, Berkshire Hathaway CEO

Berk­shire Hath­away pulls out of Ab­b­Vie, Bris­tol My­ers Squibb in­vest­ments

It looks like Warren Buffett is sticking to ice cream and railroads for the moment.

The billionaire CEO of Berkshire Hathaway backed out of two major holdings in the pharma industry, Forexlive first reported, including a $410 million investment in AbbVie and a $324.4 million stake in Bristol Myers Squibb.

The move comes after Berkshire abandoned its Teva shares just last quarter, Bloomberg reported.

Long-ex­pect­ed UK lay­offs im­mi­nent for No­var­tis fol­low­ing sale

Nearly a year ago, more than 200 workers at Novartis’ Grimsby, UK, facility were able to hang on to their jobs after the pharma closed a Switzerland site as a part of its workforce restructuring plan. Now, it looks like those employees’ time is up, as the site has been sold, Grimsby Telegraph reported today.

The manufacturing site has been sold to Humber Industrials, a subsidiary of International Process Plants. None of the current staff members will be working with the new owners, however.