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.

Regeneron Pharmaceuticals will share in development of Tessera Therapeutics’ TSRA-196, a Phase 1-ready gene-editing therapy for alpha-1 antitrypsin deficiency (AATD). Other companies developing genetic medicines for this rare disease include Beam Therapeutics, Wave Life Sciences, Korro Bio, and AIRNA.

The post Regeneron Puts Up $150M to Partner on Tessera Gene-Editing Med for Rare Liver & Lung Disorder 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.

Beam Therapeutics’ first clinical data in the rare disease alpha-1 antitrypsin deficiency “set a bar for efficacy in the space,” according to one analyst. But the ongoing market slide poses challenges for biotech companies aiming to raise money by selling stock.

The post Beam Base Editor’s Early Data in Rare Liver Disease Tee Up $500M Stock Sale in Tough Market appeared first on MedCity News.

The death wasn’t due to Beam Therapeutics’ sickle cell disease therapy, BEAM-101, but was instead attributed to the preconditioning treatment needed before the genetic medicine is infused. A different Beam program in preclinical development could avoid the need for toxic preconditioning; data from both programs will be presented during the upcoming American Society of Hematology annual meeting.

The post Beam’s Base-Editing Sickle Cell Therapy Shows Early Promise, But a Fatality Overshadows Results appeared first on MedCity News.

Editas Medicine says its ex vivo gene-editing therapy for hemoglobinopathies has best-in-class potential, but the company plans to out-license or partner that program. Editas is shifting focusing to in vivo gene-editing therapies.

The post Editas Medicine Seeks Partner for Ex Vivo Gene-Editing Program as Focus Turns to In Vivo R&D appeared first on MedCity News.

Bristol Myers Squibb is turning to the prime-editing technology of Prime Medicines for the R&D of new cell therapies for cancer and immunology. The pharma giant is paying $110 million up front to begin the collaboration.

The post Gene-Editing Biotech Prime Medicine Strikes Up Cell Therapy R&D Alliance With Bristol Myers Squibb 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.

Metagenomi finds novel gene-editing tools by mining microbial samples from around the world. Already partnered with Moderna and Ionis Pharmaceuticals, the company said its IPO cash will support ongoing preclinical research.

The post Moderna & Ionis-Partnered Biotech Lands $94M in IPO Cash for Gene-Editing R&D appeared first on MedCity News.

Eli Lilly is acquiring Beam Therapeutics’ opt-in rights to three Verve Therapeutics gene-editing therapies for cardiovascular conditions. The deal comes four months after the pharmaceutical giant began a partnership on a preclinical Verve gene-editing therapy for a different target.

The post Eli Lilly Strikes $250M Deal to Add Gene-Editing Medicines for Cardio Conditions appeared first on MedCity News.

Sickle cell disease is among the Beam Therapeutics programs spared, and here the company needs to show differentiation from genetic medicines under FDA review from Vertex Pharmaceuticals and Bluebird Bio. Meanwhile, Beam aims to find partners for its research in allogeneic cancer cell therapies and hepatitis B.

The post Base-Editing Biotech Beam Restructures, Narrowing Pipeline & Cutting 20% of Staff appeared first on MedCity News.

Imugene gains global rights to Precision BioSciences’ most advanced program, an allogeneic cell therapy for advanced cases of blood cancer. Precision will now focus on in vivo gene editing therapies, some of which is partnered with Novartis and Eli Lilly.

The post Precision Bio Offloads Lead Cell Therapy Program in Pivot to In Vivo Gene Editing appeared first on MedCity News.

Eli Lilly’s partnership with Verve Therapeutics covers the development of a preclinical therapy addressing a protein associated with cardiovascular disease risk. The in vivo gene-editing therapy is a potential one-time treatment.

The post Eli Lilly Puts Up $60M for In Vivo Gene-Editing Med for Cardiovascular Disease appeared first on MedCity News.

Vertex Pharmaceuticals aims to use CRISPR Therapeutics’ gene-editing technology to develop type 1 diabetes cell therapies that don’t prompt the immune system to reject them. This approach could compete with a Sana Biotechnology program expected to reach its first test in humans this year.

The post Vertex Pays CRISPR Therapeutics $100M to Bring Gene-Editing to Type 1 Diabetes appeared first on MedCity News.

GV led Chroma Medicine’s Series B financing round, which the biotech will apply toward epigenetic medicines that could offer advantages over other editing technologies. Other recent financings include rounds from Cargo Therapeutics and Transcend Therapeutics.

The post Aiming for an Epigenetics Edge, Chroma Raises $135M to Move Closer to the Clinic appeared first on MedCity News.

Moderna is partnering with Life Edit, a subsidiary of ElevateBio that offers a suite of gene-editing technologies. The deal brings together the capabilities of both companies, which aim to develop in vivo gene-editing therapies.Mod

The post Moderna Signs On a New Partner in Quest for In Vivo mRNA Gene Editing 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.

Verve Therapeutics received the formal FDA letter outlining what the agency wants to see before it lifts the clinical hold placed on the biotech’s gene-editing therapy. The lifting of a clinical hold on another experimental genetic medicine was among the other recent regulatory news from the past week, which included two drug approvals and one rejection.

The post FDA Tells Verve What to Do to Get Hold Lifted From Gene-Editing Therapy appeared first on MedCity News.

The FDA placed a clinical hold on Verve Therapeutics’ application to begin human testing of its gene-editing therapy for an inherited form of high cholesterol. No details about the FDA’s questions or concerns were disclosed; so far, no serious safety problems have been reported in the U.K. and New Zealand where the clinical trial is already underway.

The post FDA Pauses Verve’s Trial Plan for Gene-Editing Therapy for High Cholesterol appeared first on MedCity News.

Prime Medicine has yet to test prime editing in humans, but the biotech company garnered enough investor interest to boost the size of its IPO, raising $175 million. Prime claims its technology has key advantages over other gene-editing technologies and the IPO cash will support a pipeline that currently spans 18 programs.

The post Prime Medicine pulls off $175M IPO and sketches out plans to reach the clinic appeared first on MedCity News.

Ascidian Therapeutics is developing therapies for inherited disorders that work by editing RNA with an approach that could offer advantages over currently available genetic medicines. The biotech’s lead program is in preclinical development for Stargardt disease, a rare eye disorder caused by multiple genetic mutations.

The post Sea creature-inspired biotech Ascidian surfaces with $50M and a new way to edit RNA appeared first on MedCity News.

AstraZeneca’s LogicBio acquisition comes at a critical time for the biotech. Though LogicBio has encouraging early clinical data for its lead gene-editing therapeutic candidate, the company’s cash woes include a flagging stock price that is set to lose its listing on the Nasdaq.

The post AstraZeneca throws lifeline to gene-editing biotech LogicBio with $68M buyout appeared first on MedCity News.

Prime Medicine likens its gene-editing technology to a word processor that searches for the correct place in the genome to make an edit, replacing or repairing a wide variety of target DNA. The preclinical biotech is now spelling out its future plans with the letters I-P-O.

The post Prime Medicine spells out IPO plans for ‘word processor’ of gene editing 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.

An Intellia Therapeutics therapy that uses CRISPR to perform in vivo genomic edits has clinical data showing that the therapeutic effects continue for a year in patients. The biotech now plans to speak with regulators about the design for a pivotal clinical trial testing this therapy, a potential one-time treatment for a rare inherited disease.

The post New data for Intellia’s CRISPR therapy shows potential for one-time treatment of a rare disease appeared first on MedCity News.

Novartis is paying Precision Bio $75 million up front to begin a partnership aiming to develop in vivo gene-editing therapies for serious genetic blood disorders, such as sickle cell disease. One of the goals of the alliance is to make genetic medicines more globally accessible.

The post Novartis picks Precision Bio to bring in vivo gene editing meds to blood disorders appeared first on MedCity News.

Gene-editing medicines developer Intellia Therapeutics is paying $45 million up front to acquire Rewrite Therapeutics. The startup’s technology brings Intellia another tool for its genetic medicines toolbox, one that could enable even more precise edits.

The post Intellia’s Rewrite Therapeutics buyout brings new precision to gene editing 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.