Gene editing started 2024 with a bang in the medical sector but ended the year with a whimper. It all started with the FDA approval of the first CRSPR-based gene therapy for sickle cell anemia (SCA) called Casgevy, developed by Vertex Pharmaceuticals Inc. NASDAQ: VRTX. Sickle cell anemia is a disease in which red blood cells become sickle-shaped, 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. To treat sickled cells, CRISPR-Cas9 is used to edit genes that inhibit the production of BCL11A, which reactivates abundant fetal hemoglobin production to reduce sickling of blood cells.
CRISPR Therapeutics: assistance in the development of Kazgevi
CRISPR therapy today
CRISPR therapy
As of 4:00 pm ET
- 52 week range
- $38.20
▼
$91.10
- Target price
- $78.38
While Vertex Pharmaceuticals brought Casgevy to market, CRISPR Therapeutics AG NASDAQ: CRSP was a key collaborator in the development of a treatment that was 93.5% effective in treating SCD patients with recurrent vaso-occlusive crises (VOC). That prompted CRISPR to seek treatments for a range of inherited diseases, kicking off the gene-editing race and sending its shares to $91.10 by the first week of February 2024. Unfortunately, the stock fell and closed the year down 34% in 2024.
Cut and Paste Tool for Gene Editing
The technology is still in its infancy and extremely expensive at $2.2 million per treatment. The patient will only need one procedure. CRISPR-Cas9 consists of two parts. The guide RNA will target a specific sequence in the genome, and Cas9 will be used as molecular scissors that precisely cut out the desired sequence. It allows scientists to edit the remaining genome by changing, inserting or deleting new sequences, making CRISPR-Cas9 a cut-and-paste tool for gene editing.
Over 90% success rate and even more treatments in the future
Vertex Pharmaceutical has presented impressive long-term data on its Kasgevy treatment. 39 of 42 patients with SCD were VOD-free for at least 12 consecutive months, with a mean duration of VOD-free status of 30.9 months. The success rate for patients with transfusion-dependent beta thalassemia (TDB) was 98%, or 53 of 54. These patients had completed at least 12 consecutive months of transfusion independence with a weighted mean hemoglobin of at least 9/gdl.
CRISPR Therapeutics Stock Forecast Today
$78.38
Growth potential 86.96%Hold
Based on ratings from 19 analysts
| High forecast | $105.00 |
|---|---|
| Average forecast | $78.38 |
| Low forecast | $48.00 |
CRISPR Therapeutics Stock Forecast Details
The average duration of transfusion independence was 34.5 months, the maximum was 64.1 months. More than 45 authorized treatment centers around the world have been activated to support Kasgevy therapy, and more than 40 patients have received their first cell collections.
CRISPR Therapeutics is focused on treating cystic fibrosis. Duchenne muscular dystrophy (DMD), hemophilia A and B, HIV, hereditary retinal disease (IRD). CRISPR expects revenues from cardiovascular treatments CTX310 and CTX320 to be reported in 2025. The company has $1.9 billion in cash to last another three years until it needs to raise capital, but the company expects to be profitable by 2028. .
Intellia Therapeutics: gene editing to treat cancer and genetic diseases
Intellia Therapeutics today
Intellia Therapeutics
As of 4:00 pm ET
- 52 week range
- $11.34
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$34.87
- Target price
- US$54.94
Another leader in gene editing is Intellia Therapeutics Inc. NASDAQ:NTLAwhich specializes in treating genetic diseases, and CRISPR Therapeutics specializes in inherited blood disorders. Intellia is also using CRISPR-Cas9 treatments to address a broader range of treatments for hereditary angioedema (HAE), alpha-1 antitrypsin deficiency (AATD), and ATTD lung diseases. The company partners with a biotech giant Regeneron Pharmaceuticals Inc. NASDAQ: REGN for the treatment of transthyretin amyloidosis (ATTR) and hemophilia A/B.
Multiple Catalysts for Excellence in 2025
Intellia reported third-quarter 2024 EPS loss of $1.34, still beating the consensus estimate by 5 cents. Revenue fell 24.1% year over year to $9.1 million, beating the consensus estimate of $8.28 million. The company received IND approval from the FDA for the Phase 3 MAGNITUDE-2 trial.
Intellia Therapeutics stock forecast today
US$54.94
Growth potential 340.56%Moderate purchase
Based on ratings from 18 analysts
| High forecast | US$120.00 |
|---|---|
| Average forecast | US$54.94 |
| Low forecast | $14.00 |
Intellia Therapeutics Stock Forecast Details
The Company reported unprecedented positive Phase 2 results with NTLA-2002 as a functional treatment for HAE. Current treatment options are limited to chronic prophylactic treatment to treat seizures and on-demand therapy to control breakthrough seizures. The company is actively participating in the third phase of the HAELO study for NTLA-2002.
Intellia CEO John Leonard commented: “With three active Phase III studies expected by the end of the year, we are leading the way in in vivo CRISPR-based medicines. Intellia is ushering in a new era of medicine with the promise of functional treatments for patients suffering from HAE and treatments that may change the course of the disease in people with ATTR amyloidosis.”
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