Things are looking great for Rocket Pharmaceuticals as they have been garnering success left and right over the past few years. 

Their stocks soared high in the past year because of their research in gene therapy cures, which have successfully tested for fair treatment for severe inherited rare heart diseases.

Meanwhile, many more follow suit as biotech companies have been in the middle of their clinical trials to solve rare conditions and diseases and look for treatments through gene editing and therapy.

RELATED: Are Gene Therapies the Medicine of the Future?

Rocket Pharmaceuticals And The Future Of Gene Therapy

What is Gene Therapy?

Gene Editing In Vitro

Gene therapy is the alteration, replacement, or use of an existing gene missing from a person and putting it in place of a missing gene causing abnormality or triggering diseases.

Some examples where gene therapy is used is as follows:

  • Replacing of defective genes

  • Speed up the destruction of cancer cells

  • Revert cancer cells and make them normal cells

  • Vaccines from viral genes

  • Growth of new tissues

  • Healing of damaged tissues


In clinical trials, there have been successes in treating leukemia, hemophilia, severe combined immune deficiency, and blindness because of retinitis pigmentosa.


Although gene therapy is shaping up to be an effective treatment for different kinds of medical conditions, there have also been some mishaps and uncertainties which couldn’t be avoided.

The risks involved are the unwanted reaction in a system, wrong cells being targeted, an infection caused by a virus, and the possibility of causing a tumor.

RELATED: Cell and Gene Therapies Shatter Prior Records, with Continued Growth Expected

Latest Achievements Of Rocket Pharmaceutical

Recently, a statement from their chief executive officer Gaurav Shah, M.D. revealed the success of their lentiviral-based gene therapy programs for rare childhood disorders.

Clinical trials for FA, LAD-I, and PKD treatment turned out to be positive and successful, and that the company is shaping something big for biotechnology and its future.

“We are very excited to report positive clinical results from three of our lentiviral-based gene therapy programs at this year’s ASGCT, which show the great potential of these therapies to successfully treat FA, LAD-I, and PKD. In the case of RP-L102 for FA and RP-L201 for LAD-I, the new data advance us closer to regulatory submissions,” said Shah. “At least six out of nine patients in our FA Phase 1 and 2 trials now show evidence of engraftment, further supporting the potential of RP-L102 to serve as a hematologic treatment option for FA in the absence of cytotoxic conditioning. Although preliminary, four out of the five patients anticipated necessary for a positive trial outcome have initially met the minimum 10% MMC resistance threshold in the bone marrow on at least one occasion, including two patients at 6-months post-treatment.”

“In our Phase 1/2 trial for LAD-I, all four patients with follow-up ranging from 3 to 18 months had CD18 expression that substantially exceeded the 4-10% threshold associated with survival into adulthood and consistent peripheral blood vector copy number, further demonstrating the potential of RP-L201 to yield durable clinical benefit. All of these patients have been free of serious infections since hospital discharge following RP-L201 therapy. Lastly, data from our Phase 1 trial of RP-L301 for PKD show that both patients’ hemoglobin levels have safely normalized, with neither patient requiring red blood cell transfusions after hematopoietic reconstitution while demonstrating improving hemolysis markers. We are proud of the progress we have made across all three programs and look forward to further advancing our investigational gene therapies to offer curative treatments to patients with these devastating diseases.”

Other Companies Experiencing Success In Gene Therapy

Sharing Rocket Pharmaceuticals’ success, these companies have been using gene therapy to solve rare human conditions in the medical field.

1. CRISPR Therapeutics

CRISPR Therapeutics is a non-profit organization and a gene-editing company using their own CRISPR/Cas-9 technology, which is used for hemoglobinopathies, cancer, diabetes, and other diseases. 

2. Asklepios BioPharmaceutical

Asklepios BioPharmaceutical, Inc. (AskBio) is a company that has been studying genetic medicine for the past several decades. They have focused on Parkinson’s disease and Pompe disease, where they have almost 20 active cases combined.

3. Editas Medicine

Editas Medicine is a gene-editing company that has been the first to try CRISPR-based medicine inside a human body. Their treatment is called EDIT-101. The said technology targeted mutated genes that caused congenital blindness.

The clinical trials for their second medication, EDIT-301, are searching for treatment for sickle cell disease.

4. Homology Medicines

Homology Medicines have been using the homologous recombination approach, which repairs DNA sequences through the cells. 

This company is famous for its HMI-102, which is a treatment for phenylketonuria. This treatment reduces the levels of Phenylalanine, which is responsible for the disorder to occur.

5. Intellia Therapeutics

Intellia Therapeutics is also a gene-editing company that uses CRISPR technology to primarily looking for a treatment for transthyretin amyloidosis, also known as ATTR.

The treatment for this rare and fatal disease is in its first phase and is also being worked on by Regeneron.

6. Pfizer

In addition to creating vaccines for COVID-19, Pfizer is also working on treatments for hemophilia A and B and muscular diseases such as Duchenne muscular dystrophy. This therapy which targets mutation-driven blood diseases is in its third phase and looks to be successful.

7. Prevail Therapeutics

Prevail Therapeutics is using its gene therapy platform to look for treating neurodegenerative diseases.

The company has been making waves in the news lately as they have entered clinical trials for their two therapies: PR001, which is for Parkinson’s Disease, and PR006, which is for dementia.

These companies are looking for ways to make life for humans easier by searching for treatments and cures for rare diseases either acquired through birth or in a person’s genes. Rocket Pharmaceuticals and other biotech companies are shaping the future with their discoveries and technologies hoping to solve some of human’s infrequent medical problems.

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