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.

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.

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. 

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.

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.

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.