Gene editing started 2024 with a bang in the medical sector, but ended the year on a whim. It all started with the US Food and Drug Administration’s approval of the first CRSPR-based gene therapy for sickle cell anemia (SCA) called Casgevy, which was developed by Vertex Pharmaceutical Company Nasdaq: Vertex. Sickle cell is a disease that causes red blood cells to form into a sickle-like shape, which may block blood flow, leading to more serious complications such as pain and organ damage. It is caused by a mutation in the hemoglobin (HBB) gene. CRISPR-Cas9 acts like molecular scissors that can precisely cut out parts of the HBB genome in living cells. To treat sickle cells, CRISPR-Cas9 is used to edit genes that block the production of BCL11A, which reactivates the production of rich fetal hemoglobin to reduce blood cell clotting.
CRISPR therapeutics: helping develop Casgevy
CRISPR therapeutics today
CRISPR therapeutics
Effective 07/01/2025 at 04:00 PM ET
- 52 week range
- $38.20
▼
$91.10
- Price target
- $78.38
While Vertex Pharmaceuticals brought Casgevy to the market, CRISPR Therapeutics AG Nasdaq: CRSB He was a key collaborator in the development of the treatment, which achieved a 93.5% efficacy rate for treating SCD patients with recurrent vaso-occlusive crises (VOCs). This spurred CRISPR into the search for cures for a whole world of genetic diseases, kick-starting the gene-editing race and sending its shares to $91.10 by the first week of February 2024. Unfortunately, shares declined to close the year down 34% in Year 2024.
Cut and paste tool for gene editing
This technology is still in its infancy and is very expensive, with the cost of the treatment reaching $2.2 million. The patient will only need one treatment. CRISPR-Cas9 consists of two parts. The guide RNA will target the specific sequence within the genome, and Cas9 will be used as molecular scissors that precisely cuts the target sequence. It enables scientists to edit the remaining genome by modifying, inserting or deleting new sequences, making CRISPR-Cas9 a cut-and-paste tool for gene editing.
Success rate of more than 90% and more treatments to come
Vertex Pharmaceutical provided impressive long-term data for its Casgevy treatment. Thirty-nine of 42 patients with SCD were free of VODs 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 had at least 12 consecutive months of transfusion independence with a weighted average hemoglobin of at least 9/g liter.
CRISPR Therapeutics Stock Outlook Today
$78.38
86.96% upHe catches
Based on 19 analyst reviews
| High expectations | $105.00 |
|---|---|
| Average expectations | $78.38 |
| Low expectations | $48.00 |
CRISPR Therapeutics Stock Outlook Details
The mean duration of transfusion independence was 34.5 months, with a maximum of 64.1 months. More than 45 authorized treatment centers worldwide have been activated to support Casgevy’s treatment, and cells have been collected for the first time in more than 40 patients.
CRISPR Therapeutics is pursuing treatments for cystic fibrosis. Duchenne muscular dystrophy (DMD), hemophilia A and B, HIV, hereditary retinal disease (IRD). CRISPR expects results for its cardiovascular disease treatments CTX310 and CTX320 in 2025. The company has $1.9 billion in cash, which it can conserve for more than three more years until a capital raise is needed, but the company expects profitability by 2028. .
Intellia Therapeutics: Gene editing to treat cancer and genetic diseases
Intellia treatments today
Intellia Treatments
Effective 07/01/2025 at 04:00 PM ET
- 52 week range
- $11.34
▼
$34.87
- Price target
- $54.94
Another pioneer in the field of gene editing is Intellia Therapeutics Inc NASDAQ:NTLAwhich focuses on treating genetic diseases, while CRISPR Therapeutics focuses on genetic blood disorders. Intellia also uses CRISPR-Cas9 therapeutics to address a wide range of treatments for hereditary angioedema (HAE), alpha-1 antitrypsin deficiency (AATD), and ATTD-lung disease. The company is collaborating with the biotech giant Regeneron Pharmaceuticals Company Nasdaq:Regn For the treatment of transthyretin amyloidosis (ATTR) and hemophilia A/B treatments.
Many incentives to achieve the best results in 2025
Intellia reported a third-quarter 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 of $8.28 million. The company has received IND approval from the U.S. Food and Drug Administration (FDA) for its Phase 3 study of MAGNITUDE-2.
Intellia Therapeutics stock forecast today
$54.94
340.56% upModerate purchase
Based on 18 analyst ratings
| High expectations | $120.00 |
|---|---|
| Average expectations | $54.94 |
| Low expectations | $14.00 |
Intellia Therapeutics stock forecast details
The company announced unprecedented positive Phase 2 results for NTLA-2002 as a functional treatment for hereditary angioedema (HAE). Current treatment options are limited to chronically administered preventive therapies to manage attacks and on-demand therapies to control breakthrough attacks. The company is examining the Phase 3 HAELO study of NTLA-2002.
“With three active Phase III studies expected by the end of the year, we are leading the field of CRISPR-based drugs in vivo,” commented John Leonard, CEO of Intellia. “Intellia heralds a new era of medicine with the potential for a functional cure for patients with angioedema.” Genetics and treatment may change the course of the disease for people with ATTR amyloidosis.
Before you consider CRISPR therapeutics, you’ll want to hear this.
MarketBeat tracks the highest-rated and best-performing research analysts on Wall Street and the stocks they recommend to their clients on a daily basis. MarketBeat identified the five stocks that top analysts are quietly whispering to their clients to buy now before the broader market catches up… and CRISPR Therapeutics was not on the list.
While CRISPR Therapeutics currently has a “Hold” rating among analysts, top-rated analysts believe these 5 stocks are better buys.
View the five stocks here
Are you considering investing in Meta, Roblox, or Unity? Click the link to find out what street investors need to know about the Metaverse and public markets before making an investment.
