Intellia Therapeutics’ lonvo-z uses CRISPR to inactivate a gene in the liver to reduce levels of a protein key to the swelling attacks from the rare disease hereditary angioedema. Intellia has filed a regulatory submission for this genetic medicine, which could become the first in vivo gene-editing therapy to land FDA approval.

The post Intellia’s Data Reveal Tees Up FDA Filing for CRISPR-Based In Vivo Gene-Editing Med appeared first on MedCity News.

The FDA clinical hold covers two Phase 3 studies for nexiguran ziclumeran, or nex-z, an experimental gene-editing therapy for transthyretin amyloidosis. Nex-z uses the CRISPR gene-editing technology to inactivate the gene that codes for the protein driving this rare disease.

The post Liver Complications Lead FDA to Stop Tests of Intellia Gene-Editing Therapy for a Rare Disease appeared first on MedCity News.

Chiesi Global Rare Diseases is committing up to $115 million to begin a collaboration on Arbor Biotechnologies’ in vivo gene-editing therapy for primary hyperoxaluria type 1, an inherited liver disorder. It’s the rare disease company’s first foray into genetic medicines.

The post Chiesi’s Rare Disease Strategy Expands to Genetic Meds via Alliance With CRISPR Startup Arbor appeared first on MedCity News.

AI is no panacea, but it can have a role in turning artisanal, trial-and-error drug development into a rapid, cost-effective, data-driven process.

The post AI Meets Gene Editing: the Path to Plug-and-Play Drug Development appeared first on MedCity News.

Caribou Biosciences’ restructuring narrows the biotech’s focus to two off-the-shelf cell therapies for blood cancers, whose key data readouts have been pushed out to the second half of 2025. It’s the CRISPR-editing company’s second cash-saving restructuring in the past year.

The post CRISPR Biotech Caribou Trims Pipeline and Cuts 32% of Staff to Focus on Two Cancer Cell Therapies appeared first on MedCity News.

Regeneron Pharmaceuticals is collaborating with CRISPR technologies startup Mammoth Biosciences to develop in vivo gene-editing therapies. The deal marks the third pharmaceutical industry partnership for Mammoth, which initially focused on developing CRISPR-based diagnostics.

The post Regeneron Bets $100M That Mammoth’s CRISPR Tech Can Deliver on Next Wave of Genetic Medicines appeared first on MedCity News.

Casgevy, from Vertex Pharmaceuticals and CRISPR Therapeutics, is now approved in the United Kingdom for treating the blood disorders sickle cell disease and beta thalassemia. It’s the first regulatory approval in the world for a CRISPR-based therapy.

The post U.K. Is First to Approve a CRISPR-Based Therapy, Covering Two Blood Disorders appeared first on MedCity News.

Graphite Bio voluntarily paused a Phase 1/2 test of its gene-edited therapy for sickle cell disease after the first patient in the study developed a serious blood complication. The setback will also delay plans to reach the clinic with another genetic medicine in its pipeline.

The post Serious Side Effect Sidelines Gene-Edited Sickle Cell Therapy from Graphite Bio appeared first on MedCity News.

Sanofi is collaborating with Scribe Therapeutics to use the startup’s CRISPR technology to create cell therapies based on natural killers, a type of immune cell that has cancer-killing capabilities. The pharma giant is paying Scribe $25 million up front to kick off the deal.

The post Sanofi turns to startup Scribe to CRISPR edit new NK cell therapies for cancer appeared first on MedCity News.

Intellia Therapeutics has encouraging early clinical data for a gene-editing therapy addressing the rare swelling disorder hereditary angioedema. This therapy, the biotech’s second CRISPR-editing based treatment that performs in vivo edits, offers potential for a one-time treatment of a disease that is currently addressed by chronic oral and injectable drugs.

The post Another Intellia CRISPR therapy flashes potential for one-time, in vivo treatment appeared first on MedCity News.

Bristol Myers Squibb is partnering with Century Therapeutics, placing a $150 million bet that the biotech’s technology for engineering stem cells can produce new off-the-shelf cell therapies for cancer. If the four potential programs covered under the pact reach the market, milestone payments could bring Century more than $3 billion.

The post BMS puts $150M on the table in off-the-shelf cell therapy alliance with Century appeared first on MedCity News.

Biotech startup GenEdit is developing polymer nanoparticle technology to deliver genetic medicines, an approach intended to avoid the limitations of viral vectors. Already partnered with a clinical-stage company, it now has financial support from a big pharma giant that joined a syndicate of investors in a $26 million Series A round.

The post Aiming for better genetic medicines delivery, startup GenEdit grabs $26M appeared first on MedCity News.

Mammoth Biosciences is applying CRISPR technology to both diagnostics and therapeutics. With the new financing, CEO Trevor Martin said that the company is looking ahead toward clinical trials and perhaps partnerships with larger companies.

The post Mammoth Bio’s massive $195M financing makes the CRISPR company a unicorn appeared first on MedCity News.

Caribou Biosciences’ CRISPR approach to cell therapy sparked strong investor interest, enabling the clinical-stage biotech to upsize its IPO and raise $304 million in the biggest life sciences IPO this week. Absci, Sophia Genetics, and Cytek Biosciences also priced IPOs.

The post CRISPR biotech Caribou Biosciences’ IPO reels in $304M for next-gen cell therapy appeared first on MedCity News.

The Food and Drug Administration had previously granted an emergency use authorization for Abbott’s ID NOW point-of-care test, a decision for which the agency came under fire amid reports of the test delivering inaccurate results.

The post Sherlock, binx to develop point-of-care Covid-19 test using CRISPR technology appeared first on MedCity News.

The companies plan to apply with the Food and Drug Administration for an emergency use authorization by the end of the year. Mammoth’s technology is based on the research of CRISPR pioneer Jennifer Doudna.

The post Mammoth Biosciences, GSK partner on CRISPR-based Covid-19 diagnostic appeared first on MedCity News.

The Cambridge, Massachusetts-based company, co-founded by CRISPR pioneer Feng Zhang, said Thursday that it had received an emergency use authorization for its SARS-CoV-2 test, which works by programming a CRISPR molecule to pick up the virus’ genetic signature.

The post Feng Zhang’s Sherlock gets first-ever CRISPR nod as FDA green-lights Covid-19 test kit appeared first on MedCity News.

The companies plan to focus initially on development of two monoclonal antibody candidates, with the aim of initiating Phase II clinical development in the next three to five months.

The post GSK to make $250M equity investment in Vir Biotechnology under Covid-19 collaboration appeared first on MedCity News.

The company was founded to develop base editing, a form of gene editing that alters genes at the single-letter level rather than using the “cutting” technique typical of CRISPR/Cas9. Beam had filed to go public in September, aiming at raising $100 million.

The post Beam Therapeutics raises $180M in IPO appeared first on MedCity News.

The latest round of funding, led by Chinese investors, brings the total amount of money raised by the company, co-founded by CRISPR pioneer Jennifer Doudna, to more than $70 million.

The post Mammoth Biosciences raises $45M Series B round for CRISPR diagnostics, therapeutics development appeared first on MedCity News.

Jiankui He, who sparked controversy in 2018 with the announcement that he had used gene-editing technology on human embryos, has been sentenced to prison and fined, Chinese state media reported. His former professor at Rice University remains under investigation by the school.

The post Report: ‘CRISPR babies’ scientist gets 3 years in prison appeared first on MedCity News.

The company, officially launched in 2018 after operating in stealth mode for a year, is developing technologies to enable genome editing at the single-letter level, as opposed to the “cutting” of DNA and RNA normally used in CRISPR/Cas9.

The post Gene editing company Beam Therapeutics aims for $100M IPO appeared first on MedCity News.

Researchers using CRISPR-Cas9 gene editing on cancer cell lines found that even when certain drugs’ putative molecular targets were removed, they still killed the cancer cells, indicating their mechanisms were not understood.

The post Even when cancer drugs work, it’s not always as intended, study finds appeared first on MedCity News.

One of the four sites of the trial, in a rare form of inherited blindness, is currently listed as open for enrollment, and the companies plan to dose the first patient soon.

The post Editas, Allergan open first trial of in vivo CRISPR therapy appeared first on MedCity News.

The company, partnered with Beam Therapeutics, Verily Life Sciences and academic institutions, plans to use gene editing to tackle coronary artery disease. However, experience with gene therapy indicates cost could be a hurdle.

The post Verve Therapeutics aims to tackle heart disease through gene editing appeared first on MedCity News.

The 18 signatories emphasized that the moratorium wouldn’t constitute a ban or include gene editing for research or treatment of diseases, but called on nations to develop a framework to govern the practice.

The post Scholars call for global moratorium on human gene editing following ‘CRISPR babies’ scandal appeared first on MedCity News.

The company is developing means using CRISPR/Cas9 to edit genomes at the single-letter level, as opposed to the DNA- and RNA-cutting method usually employed.

The post Single-letter gene editing startup Beam Therapeutics raises $135M Series B round appeared first on MedCity News.

Shares of gene-editing companies were down Tuesday morning following the news, but analysts sought to reassure investors about Editas.

The post Editas Medicine CEO steps down as company moves into product development stage appeared first on MedCity News.

The company said it would use the funding to expand its single-cell DNA sequencing platform into CRISPR applications and global markets.

The post Mission Bio raises $30M in Series B round to develop genomics platform appeared first on MedCity News.

Tango Therapeutics – which uses CRISPR-based screening to find drug candidates – will receive $50 million upfront and be eligible for up to $1.7 billion in milestones, royalties and other payments.

The post Gilead Sciences, Tango Therapeutics join forces on immuno-oncology appeared first on MedCity News.