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3 Biotechs With Promising Gene Therapies in the Spotlight
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Recently, bluebird bio, Inc. (BLUE - Free Report) obtained FDA approval for its third gene therapy, lovotibeglogene autotemcel (lovo-cel), for the treatment of sickle cell disease (“SCD”) in patients aged 12 and older who have a history of vaso-occlusive events (“VOEs”). The FDA approved lovo-cel under the brand name Lyfgenia. Concurrently, it also approved Vertex Pharmaceuticals and CRISPR Therapeutics’ exagamglogene autotemcel, a CRISPR/Cas9 genome-edited cell therapy, for the treatment of SCD in patients 12 years and older with recurrent vaso-occlusive crises under the brand name Casgevy.
Casgevy is the first FDA-approved therapy utilizing CRISPR/Cas9, a type of genome editing technology where hematopoietic (blood) stem cells of patients are modified by genome editing using CRISPR/Cas9 technology. Lyfgenia is a cell-based gene therapy, which works by genetically modifying a patient’s blood stem cells to produce HbAT87Q.
However, stocks of these companies were down despite the approvals due to the uncertainty regarding the uptake of these therapies, given the high prices of these treatments and side effects. Nevertheless, this space remains in the spotlight in a volatile biotech sector, given the need for innovative treatments.
The recent FDA approval of the two gene therapies has put the focus on biotechs like Editas Medicine (EDIT - Free Report) , Beam Therapeutics Inc. (BEAM - Free Report) and Intellia Therapeutics (NTLA - Free Report) , which have promising candidates in their pipeline.
Editas, a clinical stage genome editing company, has developed a proprietary gene editing platform based on CRISPR technology. Editas’ lead program, EDIT-301, is an experimental ex vivo gene-edited medicine being evaluated for the treatment of SCD and transfusion-dependent beta thalassemia (“TDT”). In October, the FDA granted Regenerative Medicine Advanced Therapy designation to EDIT-301 for the treatment of SCD.
Enrolment and dosing of patients is ongoing in the RUBY trial for SCD. Editas also continues to enroll and dose patients in the EdiTHAL trial for TDT. The company recently announced new safety and efficacy data for 17 patients treated with EDIT-301, now known as enizgamglogene autogedtemcel (reni-cel), from both these studies.
In both the RUBY and EdiTHAL trials to date, reni-cel has been well-tolerated and continues to demonstrate a safety profile consistent with myeloablative conditioning with busulfan and autologous hematopoietic stem cell transplant in all patients. All RUBY patients are free of VOEs since treatment with reni-cel while EdiTHAL patients had early and robust increase of total hemoglobin, above the transfusion independence threshold of 9 g/dl.
The promising preliminary results underscored the potential of this gene therapy particularly in the safety aspect. The company continues dosing additional patients and will share further updates in mid-2024. Positive additional updates bode well for Editas.
Editas had cash, cash equivalents, and marketable securities of $446.4 million as of Sep 30, 2023, which it expects to use for operating expenses and capital expenditures into the third quarter of 2025. Hence, additional sources of funding are required.
Beam Therapeutics’ pipeline of gene editing technologies is based on proprietary base editing technology. This enables a differentiated class of precision genetic medicines that target a single base in the genome without making a double-stranded break in the DNA. The company is advancing its base editing technology across three disease area portfolios: hematology, immunology/oncology and genetic diseases.
BEAM is evaluating BEAM-101 in adult patients with severe SCD. The first patient in the BEACON trial, is expected to be treated with BEAM-101 shortly. The company plans to continue enrolling additional patients in the BEACON trial through the end of year and beyond, with a total target of 45 treated patients. Initial data from the BEACON trial is expected in 2024. BEAM is also advancing its two lead in vivo base editing product candidates, BEAM-302 for the treatment of alpha-1 antitrypsin deficiency and BEAM-301 for the treatment of glycogen storage disease Ia.
The company has also dosed its first patient in the phase I/II study of BEAM-201, a multiplex-edited allogeneic CAR-T product candidate, for the treatment of relapsed/refractory T-cell acute lymphoblastic leukemia /T-cell lymphoblastic lymphoma. Enrollment is ongoing and the company expects to report initial data in 2024.
In October 2023, Beam entered into a transfer agreement with Eli Lilly, pursuant to which Lilly acquired certain assets and other rights under the Verve Agreement. Following this, Beam received an upfront payment of $200 million as well as a $50 million equity investment from Lilly.
Though still in nascent stages, BEAM’s pipeline holds potential. The company currently carries a Zacks Rank #3.
Intellia Therapeutics is also a clinical-stage genome editing company, focused on developing potentially curative therapeutics using CRISPR/Cas9-based technologies. The company’s lead in vivo candidates, NTLA-2001 for the treatment of transthyretin (“ATTR”) amyloidosis and NTLA-2002 for the treatment of hereditary angioedema (“HAE”) are the first CRISPR/Cas9-based therapy candidates to be administered systemically, via intravenous infusion, for precision editing of a gene in target tissue in humans.
NTLA-2001, the first investigational CRISPR-based therapy to be systemically delivered to edit genes inside the human body, has the potential to be the first single-dose treatment for ATTR amyloidosis. This therapy, being developed with Regeneron, offers the possibility of halting and reversing the disease by driving a deep, consistent and potentially lifelong reduction in transthyretin protein (TTR) after a single dose. The company recently announced new positive interim results from the phase I study of NTLA-2001 wherein updated data from over 60 patients showed consistent, deep and durable serum TTR reduction achieved with a single dose of NTLA-2001.
NTLA-2002 is designed to knock out the kallikrein B1 gene in the liver, with the potential to permanently reduce total plasma kallikrein protein and activity, a key mediator of HAE. It is being evaluated in a phase I/II study in adults with type I or type II HAE. A late-stage study is being planned for 2024.
Other candidates include NTLA-3001 for the treatment of Alpha-1 Antitrypsin Deficiency-associated lung disease and NTLA-2003 for the treatment of AATD-associated liver disease.
Intellia also carries a Zacks Rank #3 currently.
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3 Biotechs With Promising Gene Therapies in the Spotlight
Recently, bluebird bio, Inc. (BLUE - Free Report) obtained FDA approval for its third gene therapy, lovotibeglogene autotemcel (lovo-cel), for the treatment of sickle cell disease (“SCD”) in patients aged 12 and older who have a history of vaso-occlusive events (“VOEs”). The FDA approved lovo-cel under the brand name Lyfgenia. Concurrently, it also approved Vertex Pharmaceuticals and CRISPR Therapeutics’ exagamglogene autotemcel, a CRISPR/Cas9 genome-edited cell therapy, for the treatment of SCD in patients 12 years and older with recurrent vaso-occlusive crises under the brand name Casgevy.
Casgevy is the first FDA-approved therapy utilizing CRISPR/Cas9, a type of genome editing technology where hematopoietic (blood) stem cells of patients are modified by genome editing using CRISPR/Cas9 technology. Lyfgenia is a cell-based gene therapy, which works by genetically modifying a patient’s blood stem cells to produce HbAT87Q.
However, stocks of these companies were down despite the approvals due to the uncertainty regarding the uptake of these therapies, given the high prices of these treatments and side effects. Nevertheless, this space remains in the spotlight in a volatile biotech sector, given the need for innovative treatments.
The recent FDA approval of the two gene therapies has put the focus on biotechs like Editas Medicine (EDIT - Free Report) , Beam Therapeutics Inc. (BEAM - Free Report) and Intellia Therapeutics (NTLA - Free Report) , which have promising candidates in their pipeline.
Editas, a clinical stage genome editing company, has developed a proprietary gene editing platform based on CRISPR technology. Editas’ lead program, EDIT-301, is an experimental ex vivo gene-edited medicine being evaluated for the treatment of SCD and transfusion-dependent beta thalassemia (“TDT”). In October, the FDA granted Regenerative Medicine Advanced Therapy designation to EDIT-301 for the treatment of SCD.
Enrolment and dosing of patients is ongoing in the RUBY trial for SCD. Editas also continues to enroll and dose patients in the EdiTHAL trial for TDT. The company recently announced new safety and efficacy data for 17 patients treated with EDIT-301, now known as enizgamglogene autogedtemcel (reni-cel), from both these studies.
In both the RUBY and EdiTHAL trials to date, reni-cel has been well-tolerated and continues to demonstrate a safety profile consistent with myeloablative conditioning with busulfan and autologous hematopoietic stem cell transplant in all patients. All RUBY patients are free of VOEs since treatment with reni-cel while EdiTHAL patients had early and robust increase of total hemoglobin, above the transfusion independence threshold of 9 g/dl.
The promising preliminary results underscored the potential of this gene therapy particularly in the safety aspect. The company continues dosing additional patients and will share further updates in mid-2024. Positive additional updates bode well for Editas.
Editas had cash, cash equivalents, and marketable securities of $446.4 million as of Sep 30, 2023, which it expects to use for operating expenses and capital expenditures into the third quarter of 2025. Hence, additional sources of funding are required.
Shares of the company have gained 10.9% year to date. Editas currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
Image Source: Zacks Investment Research
Beam Therapeutics’ pipeline of gene editing technologies is based on proprietary base editing technology. This enables a differentiated class of precision genetic medicines that target a single base in the genome without making a double-stranded break in the DNA. The company is advancing its base editing technology across three disease area portfolios: hematology, immunology/oncology and genetic diseases.
BEAM is evaluating BEAM-101 in adult patients with severe SCD. The first patient in the BEACON trial, is expected to be treated with BEAM-101 shortly. The company plans to continue enrolling additional patients in the BEACON trial through the end of year and beyond, with a total target of 45 treated patients. Initial data from the BEACON trial is expected in 2024. BEAM is also advancing its two lead in vivo base editing product candidates, BEAM-302 for the treatment of alpha-1 antitrypsin deficiency and BEAM-301 for the treatment of glycogen storage disease Ia.
The company has also dosed its first patient in the phase I/II study of BEAM-201, a multiplex-edited allogeneic CAR-T product candidate, for the treatment of relapsed/refractory T-cell acute lymphoblastic leukemia /T-cell lymphoblastic lymphoma. Enrollment is ongoing and the company expects to report initial data in 2024.
In October 2023, Beam entered into a transfer agreement with Eli Lilly, pursuant to which Lilly acquired certain assets and other rights under the Verve Agreement. Following this, Beam received an upfront payment of $200 million as well as a $50 million equity investment from Lilly.
Though still in nascent stages, BEAM’s pipeline holds potential. The company currently carries a Zacks Rank #3.
Intellia Therapeutics is also a clinical-stage genome editing company, focused on developing potentially curative therapeutics using CRISPR/Cas9-based technologies. The company’s lead in vivo candidates, NTLA-2001 for the treatment of transthyretin (“ATTR”) amyloidosis and NTLA-2002 for the treatment of hereditary angioedema (“HAE”) are the first CRISPR/Cas9-based therapy candidates to be administered systemically, via intravenous infusion, for precision editing of a gene in target tissue in humans.
NTLA-2001, the first investigational CRISPR-based therapy to be systemically delivered to edit genes inside the human body, has the potential to be the first single-dose treatment for ATTR amyloidosis. This therapy, being developed with Regeneron, offers the possibility of halting and reversing the disease by driving a deep, consistent and potentially lifelong reduction in transthyretin protein (TTR) after a single dose. The company recently announced new positive interim results from the phase I study of NTLA-2001 wherein updated data from over 60 patients showed consistent, deep and durable serum TTR reduction achieved with a single dose of NTLA-2001.
NTLA-2002 is designed to knock out the kallikrein B1 gene in the liver, with the potential to permanently reduce total plasma kallikrein protein and activity, a key mediator of HAE. It is being evaluated in a phase I/II study in adults with type I or type II HAE. A late-stage study is being planned for 2024.
Other candidates include NTLA-3001 for the treatment of Alpha-1 Antitrypsin Deficiency-associated lung disease and NTLA-2003 for the treatment of AATD-associated liver disease.
Intellia also carries a Zacks Rank #3 currently.