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Takeda's trans­la­tion­al cell ther­a­py group revs up for a race to the clin­ic with off-the-shelf CAR-T

Four years after Takeda launched a wide-ranging induced pluripotent stem cell project with the researchers at Nobel prize-winning Shinya Yamanaka’s lab at the University of Kyoto, the pharma company is taking delivery of the first of what it hopes will be a whole pipeline of iPS cell-derived therapies that can deliver on the promise of off-the-shelf CAR-T therapies.

From here, Stefan Wildt — the head of pharma sciences and translational cell therapy at Takeda — and his group of 100-plus scientists will be charged with steering their way to the clinic as they build out the manufacturing and support work for this pipeline-in-the-making. 

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Mi­cro­cap Capri­cor soars on in­ter­im PhII DMD da­ta show­ing func­tion­al ben­e­fit for old­er pa­tients

With the interim analysis for its Phase II Duchenne muscular dystrophy, little Capricor Therapeutics was merely looking for a guidepost, a signal, a trend that the effects seen in an earlier trial can be replicated with a new dosing regimen and delivery method. Instead, the biotech found itself blessed with a basket of data points that seem to buck the natural history trend and separate the drug from placebo in a statistically significant way, more than doubling its tiny stock $CAPR in pre-market trading.

Darrell Irvine. MIT

Su­per­charg­ing CAR-T with can­cer vac­cine, MIT team spot­lights some new tech un­der­pin­ning Dar­rell Irvine's start­up

Many of the efforts to improve on the first generation of CAR-T therapies such that they can reach solid tumors had focused on tweaks inherent to the cancer killing agent — specifically, utilizing more potent T cells as their base, from stem memory T cells to virally associated T cells to marrow infiltrating lymphocytes. But what if just amplifying CAR-T cells can do the job? Darrell Irvine and his team at MIT have some intriguing mouse data for one such tech.

Christi Shaw at JP Morgan 2019. Jeff Rumans for Endpoints News

Fresh out of Eli Lil­ly, Christi Shaw sur­faces as Daniel O'­Day's new CEO at CAR-T pi­o­neer Kite

Well, that didn’t take long. 

We found out Thursday evening that Christi Shaw has given up her top post as the head of the Bio-Medicines group at Eli Lilly for the helm at CAR-T pioneer Kite. New Gilead CEO Daniel O’Day, a Roche veteran, had made finding a Kite CEO a top priority on his arrival at Gilead. And he went right for a headliner.

O’Day was clearly excited about the coup.

“We conducted an extensive search for a new leader at Kite and we believe that Christi’s unique set of skills will allow us to continue to build on our leadership position in cell therapy,” he said in a prepared statement. “Christi’s vast experience across complex therapeutic areas, and particularly in oncology, will serve Kite very well. She is clearly a leader who will bring teams and individuals together and I am confident she will build upon the entrepreneurial spirit at Kite as we seek to help more people with cancer around the world.”

Jean-Jacques Bienaimé. BioMarin via YouTube

Bil­lions on the line: Bio­Marin bus­tles past en­thused ri­val, plan­ning to launch world’s first he­mo­phil­ia A gene ther­a­py

BioMarin isn’t going to let a rival’s success over the weekend get in the way of making a bee-line to the FDA with an imperfect, but presentable, gene therapy for hemophilia A.

On Monday the biotech $BMRN reported that its talks with the FDA and the EMA convinced execs to make a leap for an accelerated approval with data on just a handful of patients. And they’re handing over Phase I/II and interim Phase III results in Q4 to make their case.

Harpreet Singh. Allogeneic Cell Therapies Summit

The chief sci­en­tist at Ger­man/US biotech hy­brid Im­mat­ics is mov­ing to the helm. Here's his to-do list

Just a few months after launching their 4th clinical trial for a unique brand of cell therapies, the German-US hybrid biotech Immatics is promoting the biotech’s long-time chief scientist and co-founder to the helm.

Harpreet Singh, CSO and head of the Houston branch of the company — which is allied with investigators at MD Anderson — has been promoted to overall CEO, a post which gives him oversight of a growing slate of trials for adoptive T cell therapies, bispecifics with a group of partners that includes Amgen, Roche and Genmab as well as an off-the-shelf approach to these new cancer therapies. 

Sang­amo/Pfiz­er are catch­ing up to Bio­Marin and Spark with a gene ther­a­py for he­mo­phil­ia A — can they leapfrog ahead?

Sangamo researchers and their partners at Pfizer may be playing catch-up with BioMarin and Spark/Roche in developing a gene therapy to cure hemophilia A, but they’re off to an encouraging start.

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Carlo Rizzuto, Versant

Bay­er, Ver­sant back a $250M cell ther­a­py start­up on a quest to cre­ate off-the-shelf im­muno-on­col­o­gy ther­a­pies

Look out Fate Therapeutics — Versant Ventures’ Century Therapeutics is lurking in the burgeoning field of off-the-shelf cell therapeutics that offer the promise of quicker, cheaper and potent cancer treatments. The Philadelphia-based biotech unveiled on Monday a meaty $250 million round of financing — led by Bayer’s venture arm — after Versant spawned it in 2018 to develop induced pluripotent stem cells (iPSC)-derived products to fight cancer.

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Novavax site in Gaithersburg, MD. Novavax

Beef­ing up its new gene ther­a­py unit, Catal­ent inks $18M deal to snap up No­vavax fa­cil­i­ties

Catalent’s hunt for rapid growth on the gene therapy front — cemented with a $1.2 billion acquisition of Paragon Biosciences two months ago — has led them to the vaccine makers at Novavax.

With an $18 million payment, Paragon is taking over two Novavax sites in Gaithersburg, MD, including more than 100 of the employees already working there. That’s in addition to a significant reduction in operating costs, says the company, as Novavax shifts to rely on Paragon for GMP materials in clinical trials and, eventually, commercial supply of their products.

Af­ter rais­ing $158M, this up­start's founders have star back­ers and plans to break new ground in gene ther­a­py

Back in 2014, Stephanie Tagliatela opted to take an early exit out of her PhD program after working in Mark Bear’s lab at MIT, where she specialized in the synaptic connections between neuronal cells in the brain. She never finished that PhD, but she and fellow MIT student Kartik Ramamoorthi — who was on the founding team at Voyager — came away with some ideas for a gene therapy startup.

Today, fully 5 years later, she and Ramamoorthi are taking the wraps off of a $104 million mega-round designed to take the cumulative work of their preclinical formative stage for Encoded Therapeutics into human studies. They’ve now raised $158 million since starting out in Illumina’s incubator in the Bay Area, and they believe they are firmly on track to do something unique in gene therapy.

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