Promising early data continue to roll out for Intellia Therapeutics Inc.’s hereditary angioedema (HAE) candidate, NTLA-2002, with one of the earliest treated patients in the phase I study remaining attack-free for more than a year. But it was the systemic CRISPR candidate’s potential as a one-time treatment that generated the most discussion on the company’s call as investors tried to assess its potential advantage in a crowded HAE market.

Bearish investors dwelling on a single grade 4 liver enzyme elevation seemed to be the cause for Intellia Therapeutics Inc.’s sinking stock Sept. 16, despite the company reporting impressive, though early stage, data for its leading systemically administered CRISPR candidates targeting hereditary angioedema (HAE) and amyloid transthyretin (ATTR) amyloidosis.

Ocular gene therapy firm Sparingvision SA raised €75 million (US$75 million) in a series B round to fund its transition to clinical development. The company is about to move its lead program, the mutation-agnostic gene therapy SPVN-06, into a phase I trial in retinitis pigmentosa (RP). “We are in the middle of the regulatory submission process,” CEO Stéphane Boissel told BioWorld.

The U.S. Patent Trial and Appeal Board’s (PTAB) decision Feb. 28 that Broad Institute scientists were the first to invent the use of CRISPR/Cas9 genome editing in eukaryotic cells is just another chapter in the ongoing saga of who has patent rights to various elements of the CRISPR platform.

CEO John Leonard said Intellia Therapeutics Inc. plans “to share information on a cohort-by-cohort basis, so we get a consistent readout” and, as the year goes on, longer-term follow-up findings will emerge from the phase I trial with the company’s lead in vivo genome editing candidate, NTLA-2001.

Intellia Therapeutics Inc. CEO John Leonard said the deal with Blackstone Life Sciences and Cellex Cell Professionals GmbH will create a new CAR T-cell therapy enterprise that bears “a German background with a strong American accent.” Blackstone committed $250 million to launch an autologous and allogeneic universal CAR T-cell therapy firm that will put together Intellia’s allogeneic cell platform plus CRISPR cell engineering with expertise from CAR T specialist Gemoab GmbH, a subsidiary of Cellex.

Advances lately in the genome-editing space include Beam Therapeutics Inc. publication in The CRISPR Journal details of its work with inlaid base editors, which the firm is applying in the BEAM-102 program for sickle cell disease. IBEs’ predictable, shifted editing window lets researchers go after disease-causing mutations that canonical base editors cannot reach, Beam said, and do the job with high efficiency and few off-target effects on the genome. The hottest news due in the near-term future from the sector will spill from Intellia Therapeutics Inc., of Cambridge, Mass., which is due to roll out first-in-human data with a systemic CRISPR-based genome editing therapy, NTLA-2001, in hereditary transthyretin amyloidosis.