Gene-Editing Stocks to Watch in 2025

Gene-editing started with a bang in 2024 Medical sector But the year closed with a whimper. This began with the FDA approval of the first CRSPR-based gene therapy treatment for sickle cell anemia (SCA), called CasGV, developed by Vertex Pharmaceuticals Inc. NASDAQ: VRTX. Sickle cell is a disease that causes red blood cells to form a sickle-like shape, which can block blood flow, leading to more serious complications such as pain and organ damage. It is caused by a mutation in the hemoglobin gene (HBB). CRISPR-Cas9 acts as molecular scissors that can precisely cut parts of the HBB genome in living cells. For the treatment of sickle cells, CRISPR-Cas9 is used Modify the genes which inhibits the production of BCL11A, which reactivates the abundant production of fetal hemoglobin to reduce blood cell disease.

CRISPR therapeutics: helping to develop casgavi

While Vertex Pharmaceuticals brought Casgavi to market, CRISPR Therapeutics AG NASDAQ: CRSP was a major contributor to the development of the treatment, which had a 93.5% efficacy rate for the treatment of SCD patients with recurrent vaso-occlusive crises (VOCs). It pushed CRISPR to treat an entire universe of hereditary diseases, kickstarting the gene-editing race and sending its shares soaring to $91.10 by the first week of February 2024. Unfortunately, the shares fell 34% in the year 2024.

Cut and paste tool for gene-editing

The technology is still in its infancy and $2.2 million a treatment is very expensive. A patient will need only one treatment. CRISPR-Cas9 consists of two parts. A guide RNA would target specific sequences within the genome, and Cas9 would be used as molecular scissors that would precisely cut the target sequence. This enables scientists to edit the rest of the genome by modifying, inserting or deleting new sequences, making CRISPR-Cas9 a cut-and-paste tool for gene editing.

Over 90% success rate and more treatments to come

Vertex Pharmaceuticals presented impressive long-term data for its Casgavi treatment. Thirty-nine of 42 patients with SCD were free of VOD for at least 12 consecutive months, with a mean duration of VIC-free status of 30.9 months. The success rate for patients with transfusion-dependent beta-thalassemia (TDT) was 98% or 53 out of 54. These patients achieve at least 12 months of continuous transfusion independence with a weighted average hemoglobin of at least 9/gdL.

The median duration of transfusion independence was 34.5 months, with a maximum of 64.1 months. More than 45 authorized treatment centers around the world have been activated to support CasGavi therapy, and more than 40 patients have had their first cell collections.

CRISPR Therapeutics is treating cystic fibrosis. Duchenne Muscular Dystrophy (DMD), Haemophilia A and B, HIV, Inherited Retinal Disease (IRD). CRISPR expects to have top-line results from its cardiovascular disease treatments CTX310 and CTX320 in 2025. The company has $1.9 billion in cash, which could sustain it for three more years unless a capital increase is needed, but the company expects to be profitable by 2028. .

Intelia Therapeutics: Gene-editing to treat cancer and genetic diseases

Another leader in gene editing Intellia Therapeutics Inc. NASDAQ: NTLAwhich focuses on treating genetic diseases, while CRISPR Therapeutics focuses on hereditary blood disorders. Intellia also uses CRISPR-Cas9 therapies to address a broad pipeline of therapies for hereditary angioedema (HAE), alpha-1 antitrypsin deficiency (AATD), and ATTD-lung disease. The company is collaborating with the biotech giant Regeneron Pharmaceuticals Inc. NASDAQ: REGN for transthyretin amyloidosis (ATTR) and hemophilia A/B treatments.

Many catalysts for top-line results in 2025

Intellia reported Q3 2024 EPS loss of $1.34, which still beat the consensus estimate by 5 cents. Revenue fell 24.1% year-over-year to $9.1 million, beating consensus estimates for $8.28 million. The company received IND clearance from the FDA for its MAGNITUDE-2 Phase 3 study.

The company reported exceptionally positive Phase 2 results for NTLA-2002 as a functional treatment for HAE. Current treatment options are limited to chronically administered prophylactic therapies to manage attacks and on-demand therapies to successfully control attacks. The Company is actively screening for the Phase 3 HAELO study for NTLA-2002.

Intellia CEO John Leonard commented, “With three active Phase III studies by the end of the year, we are leading the way in in vivo CRISPR-based medicine. Intellia is ushering in a new era of medicine with the potential for a functional treatment for patients suffering from HAE and a treatment that can change the course of the disease for people with ATTR Amyloidosis.”

Before considering CRISPR therapeutics, you may want to hear this.

MarketBeat keeps track of Wall Street’s top-rated and best-performing research analysts and the stocks they recommend to their clients on a daily basis. MarketBeat has identified Five stocks That top analysts are quietly telling their clients to buy now before the wider market hits… and CRISPR therapeutics wasn’t on the list.

While CRISPR Therapeutics currently has a “hold” rating among analysts, top analysts believe these five stocks are better buys.

Check out five stocks here

Like this article? Share it with a friend.

Link copied to clipboard.