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Reg­u­la­tors throw up an­oth­er hur­dle to Roche’s $4.3B Spark ac­qui­si­tion as ques­tions per­co­late over he­mo­phil­ia mar­ket dom­i­na­tion

Don’t look for Roche’s $4.3 billion Spark $ONCE buyout — announced 7 months ago — to close anytime in the very near future.

The UK Competition and Markets Authority just opened a 2-week public comment period on the tie-up, looking for some opinions on the pharma giant’s competitive position if the deal goes through. 

In an announcement today the CMA remarked that they were soliciting remarks on “whether the creation of that situation may be expected to result, in a substantial lessening of competition within any market or markets in the United Kingdom for goods or services.”

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No­var­tis ac­cus­es 2 AveX­is founders of al­ter­ing da­ta, stonewalling probe — but ex-CSO blasts back

Novartis has responded to the FDA’s questions on the AveXis data manipulation scandal, blaming two “senior” execs at AveXis who had helped found the company and remained “fixtures” at the biotech for their role in stonewalling the probe and delaying their response. And the pharma giant promised that it would ramp up new plans to retrain AveXis staff and accelerate an integration plan to the global organization to make sure it never happened again.

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Zol­gens­ma did­n't cause an in­fant death, AveX­is ex­ecs say as they spot­light long-term da­ta

Before the recent data manipulation scandal engulfed Novartis’ star gene therapy Zolgensma, the death of an infant in one of their Phase III trials threatened to sound safety alarms about the spinal muscular atrophy treatment. But scientists at the AveXis subsidiary now say an investigation has cleared that particular worry.

Nestled in a lengthy presentation of upbeat data, the update offers some much-needed validation for Zolgensma as execs scramble to rebuild their credibility with both regulators and the general public.

David Grainger [file photo]

'Dis­con­nect the bas­tard­s' — one biotech's plan to break can­cer cell­s' uni­fied de­fens­es

Chemotherapy and radiotherapy are the current gladiators of cancer treatment, but they come with well-known limitations and side-effects. The emergence of immunotherapy — a ferocious new titan in oncologist’s toolbox — takes the brakes off the immune system to kill cancer cells with remarkable success in some cases, but the approach is not always effective. What makes certain forms of cancer so resilient? Scientists may have finally pieced together a tantalizing piece of the puzzle, and a new biotech is banking on a new approach to fill the gap.

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Ear­ly snap­shot of Ad­verum's eye gene ther­a­py sparks con­cern about vi­sion loss

An early-stage update on Adverum Biotechnologies’ intravitreal gene therapy has triggered investor concern, after patients with wet age-related macular degeneration (AMD) saw their vision deteriorate, despite signs that the treatment is improving retinal anatomy.

Adverum, on Wednesday, unveiled 24-week data from the OPTIC trial of its experimental therapy, ADVM-022, in six patients who have been administered with one dose of the therapy. On average, patients in the trial had severe disease with an average of 6.2 anti-VEGF injections in the eight months prior to screening and an average annualized injection frequency of 9.3 injections.

Luigi Naldini, Pierluigi Paracchi, Carlo Russo. Genenta

Ital­ian gene ther­a­py play­er draws new in­vestor from Chi­na for its an­swer to CAR-T re­laps­es

So you’ve been treated with one of the new-age cancer therapies. What do you do if there’s a relapse and the tumors returns? 

Genenta Science launched four years ago out of Milan in part to answer that question, and today they received $14.4 million in round three funding that will propel their unique gene therapy through Phase I/II trials for multiple myeloma and glioblastoma. Backed now by money from the Chinese firm Qianzhan Investment Management and Fidim, the former owner of the biopharmaceutical Rottapharm, the company is also exploring ways of delivering gene therapy to solid tumors that have been harder to reach. 

Javon Ayers and his mother, Shakara Gross

A new play­er jumps in­to the heat­ed race for a sick­le cell cure, with help from Google's GV

For 17 years, Javon Ayers lived the life of most sickle cell kids: constant hospital visits, hand and foot syndrome, pneumonia and a removed spleen. In 2015, the family moved from Chicago to Georgia, which helped. 

It proved a lucky break. This year, a doctor at the Children’s Hospital of Atlanta told him he may be eligible for an experimental gene therapy that could completely cure him. What followed was a NASA-level battery of tests, a bone marrow extraction-treatment-and-reinjection, chemotherapy and three months post-op in the hospital.

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Dmitry Kuzmin, Owen Smith. 4BIO

Lon­don's 4BIO Cap­i­tal un­veils first close of $150M fund ded­i­cat­ed to ad­vanced ther­a­pies

Orchard $ORTX. Seres $MCRB. RetroSense (acquired by Allergan). With several high profile exits under their belt, 4BIO Capital is out to find more trailblazers to back in the drug R&D world.

The VC shop has raised $50 million in the first close of what it hopes to be a $150 million second fund at the end of next year. With a focus on advanced therapies — cell and gene therapy, RNA-based therapy, targeted therapy, and the microbiome — they will devote 60% of the fund to European deals from their perch in London, while spreading the rest between Japan and the US, going into the Midwest and the South for promising ideas that might not have received as much love as those coming out of Boston.

Plot­ting clin­i­cal en­try, James Wilson's gene ther­a­py start­up brings in bil­lion­aire for $110M Se­ries B

The gene therapy biotech that James Wilson helped found to take some rare disease programs out of Penn all the way to an approval has scored $110 million in its latest financing.

Passage Bio is getting a boost just seven months after launching with another megaround. Access Biotechnology — the therapeutic-focused investment arm of billionaire Len Blavatnik’s conglomerate — is leading the Series B, which also included all the marquee investors who provided the initial $115 million: OrbiMed, Frazier Healthcare Partners, Versant Ventures, Lily Asia Ventures, New Leaf Venture Partners and Vivo Capital. New backers include Boxer Capital of Tavistock Group, Highline Capital Management, Logos Capital and Sphera Funds Management.

Paul Hastings. Nkarta

Nkar­ta maps out clin­i­cal, man­u­fac­tur­ing plans for CAR-NK fol­low­ing $114M round led by Sam­sara

Back in 2015 NEA Ventures, SR One and Novo Holdings pulled together $11 million to kickstart the latest venture to come out of Dario Campana’s lab — which has also birthed Unum and MediSix — focused on natural killer (NK) cells. Four stealthy years later, Nkarta is poised for the clinic with a $114 million Series B to fuel the big leap.

While the current buzz on cell therapies for cancer has generally centered around some variation of T cells from the approved CAR-T to TCR, Nkarta believes NK cells offer advantages that T cells lack. Since they are part of the innate immune system, NK cells can identify and hit a broader range of targets presented on tumor cells.