Gene-editing Company Intellia Therapeutics Files for a $120 Million IPO

Engineered Human Therapies
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April 20, 2016

On Monday April 11th, Intellia Therapeutics filed for an IPO to raise up to $120 million.

Founded in 2014, Intellia Therapeutics (to be Nasdaq: NTLA) is focused on developing both in-vivo and ex-vivo gene therapies using CRISPR/Cas9 technology. The firm’s founders include Jennifer A. Doudna, with her intellectual property claims regarding the use of the CRISPR/Cas9 system, licensed through Caribou Biosciences. Intellia is based in Cambridge, MA and led by CEO Nessan Bermingham. The company first filed a confidential draft of registration for IPO back in September of 2015. Intellia has not yet named the number of shares to be sold, or the IPO share price, though the SEC filing provides new information on costs, revenue, and company goals.An IPO 2 years after the founding is early for a biotech company, but expected in this case, particularly given the IPO of competitor Editas Medicine(NASDAQ: EDIT) in February. Editas represents an opposing force in a highly public and ongoing intellectual property battle over aspects of CRISPR/Cas9 technology. In February, Editas raised $94m in its IPO closing at 16$ per share, following $210 million in private funding. It is now trading at just under $40 per share. On Editas IPO date, February 2nd, the Nasdaq biotechnology index (NASDAQ: NBI) was at 2727, today it's at 2936, 7.7% higher, though still well below its peak in July of 4165.Intellia, Jennifer Doudna, UC Berkeley, and Caribou Biosciences represent one side of the patent battle, while Editas, The Broad Institute at MIT, and Feng Zhang represent the other. You can view the Broad Institute patent here, and the interference request by Doudna et al. here. Notably, both firms emphasize the risks of intellectual property disputes in their SEC filings, including that high litigation costs present risk regardless of the legal outcome.Private equity funding of Intellia totaled $85 million in two rounds-- $15m in 11/2014, $70m in 09/2015, led by Atlas Ventures and OrbiMed HealthCare Fund Management respectively. Both rounds included strategic investment from Novartis. Intellia has also received $19m from collaboration with Novartis, totaling $104m in fundraising. Additionally, Intellia stands to receive a $75m payment from its Regeneron collaboration. Operating losses totalled $21.9m from 2014 inception to the end of 2015 and Intellia expects costs to rise as it enters the public market and works to advance candidate products. All revenue thus far has derived from collaborations with Novartis and Regeneron.Given the intense patent battle, the large fundraising amounts, and the early IPOs of both companies, it's well worth asking what and who are driving the intensity. It is more than a fight between the businesses—in fact, the firms may be wholly secondary to the intellectual property battle. Perhaps the fundraising by both Editas and Intellia is in order to gain leverage to cause a favorable settlement. If so, one would expect the competition to continue intensely as the firms push to be first to market. Another factor is the role of the universities in the IP dispute—MIT and UC Berkeley are two immensely powerful institutions with stakes in the outcome.Let’s recall what is at stake: if CRISPR/Cas9 remains the best genetic engineering tool available for the duration of the patent, then whoever owns the intellectual rights to CRISPR based therapeutics will control the next 20 years of gene editing therapeutics. Such therapeutics—single treatment cures to genetic based diseases-- could immensely improve human health.However, it is worth remembering that Transcription Activator Like Endonucleases (TALENs) and Zinc-Finger Nucleases (ZFNs) were each once the best available tool, now seen as old news compared with CRISPR. It is plausible that the awareness and value generated by CRISPR/Cas9 will spur greater interest in and funding of molecular biology research, resulting in the production of new discoveries and intellectual property that devalues CRISPR Cas systems. Alternatively, modifications to the CRISPR/Cas9 system may differentiate it sufficiently to create new intellectual property.In the SEC filing, Intellia described its general intended use of funds from the IPO as to advance current candidate products through R&D, submit one or more INDs (Investigational New Drug Application), develop additional in-vivo and ex-vivo product candidates, and further develop its CRISPR/Cas9 gene editing technology.Intellia indicates that its future profitability, among other factors, depends largely on further developing its CRISPR/Cas9 technology, negotiating collaborations favorably, selecting and investing in the right product candidates, advancing them through clinical trials and regulatory approval followed by commercializing and scaling, and protecting and expanding its intellectual property portfolio.Intellia’s process for selecting the best target therapies is to evaluate the type of edit-knockout, repair or insertion, the delivery modality, the presence of established therapeutic endpoints, and the potential for a CRISPR/Cas9 therapy to offer greater benefit than existing technology like TALENs or ZFNs.The company’s five-year collaboration with Novartis began in December of 2014 to develop ex vivo therapies using CRISPR/Cas9 to edit chimeric antigen receptor T cells (CAR T-cells) and Hematopoietic stem cells (HSCs). Intellia is entitled to receive $20 million in technology access fees, $20 million in research payments, and can earn development, regulatory, and sales milestone payments.Just this month, Intellia entered a licensing deal and collaboration with Regeneron to work on gene edits in the liver as well as joint research and development to improve the CRISPR/Cas system. Intellia stands to receive a $75 million payment and, on a per-license basis, can earn payments for development, regulatory, and sales-based milestones.Beyond the work with Novartis and Regeneron, Intellia’s proprietary work includes alpha-1 antitrypsin deficiency, hepatitis B virus, and inborn errors of metabolism.In selecting product candidates, advancing the proprietary and collaborative components of its pipeline, managing the costs of future scaling and commercialization, and protecting its intellectual property, Intellia faces substantial long term challenges with great potential reward.

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Gene-editing Company Intellia Therapeutics Files for a $120 Million IPO

by
April 20, 2016

Gene-editing Company Intellia Therapeutics Files for a $120 Million IPO

by
April 20, 2016

On Monday April 11th, Intellia Therapeutics filed for an IPO to raise up to $120 million.

Founded in 2014, Intellia Therapeutics (to be Nasdaq: NTLA) is focused on developing both in-vivo and ex-vivo gene therapies using CRISPR/Cas9 technology. The firm’s founders include Jennifer A. Doudna, with her intellectual property claims regarding the use of the CRISPR/Cas9 system, licensed through Caribou Biosciences. Intellia is based in Cambridge, MA and led by CEO Nessan Bermingham. The company first filed a confidential draft of registration for IPO back in September of 2015. Intellia has not yet named the number of shares to be sold, or the IPO share price, though the SEC filing provides new information on costs, revenue, and company goals.An IPO 2 years after the founding is early for a biotech company, but expected in this case, particularly given the IPO of competitor Editas Medicine(NASDAQ: EDIT) in February. Editas represents an opposing force in a highly public and ongoing intellectual property battle over aspects of CRISPR/Cas9 technology. In February, Editas raised $94m in its IPO closing at 16$ per share, following $210 million in private funding. It is now trading at just under $40 per share. On Editas IPO date, February 2nd, the Nasdaq biotechnology index (NASDAQ: NBI) was at 2727, today it's at 2936, 7.7% higher, though still well below its peak in July of 4165.Intellia, Jennifer Doudna, UC Berkeley, and Caribou Biosciences represent one side of the patent battle, while Editas, The Broad Institute at MIT, and Feng Zhang represent the other. You can view the Broad Institute patent here, and the interference request by Doudna et al. here. Notably, both firms emphasize the risks of intellectual property disputes in their SEC filings, including that high litigation costs present risk regardless of the legal outcome.Private equity funding of Intellia totaled $85 million in two rounds-- $15m in 11/2014, $70m in 09/2015, led by Atlas Ventures and OrbiMed HealthCare Fund Management respectively. Both rounds included strategic investment from Novartis. Intellia has also received $19m from collaboration with Novartis, totaling $104m in fundraising. Additionally, Intellia stands to receive a $75m payment from its Regeneron collaboration. Operating losses totalled $21.9m from 2014 inception to the end of 2015 and Intellia expects costs to rise as it enters the public market and works to advance candidate products. All revenue thus far has derived from collaborations with Novartis and Regeneron.Given the intense patent battle, the large fundraising amounts, and the early IPOs of both companies, it's well worth asking what and who are driving the intensity. It is more than a fight between the businesses—in fact, the firms may be wholly secondary to the intellectual property battle. Perhaps the fundraising by both Editas and Intellia is in order to gain leverage to cause a favorable settlement. If so, one would expect the competition to continue intensely as the firms push to be first to market. Another factor is the role of the universities in the IP dispute—MIT and UC Berkeley are two immensely powerful institutions with stakes in the outcome.Let’s recall what is at stake: if CRISPR/Cas9 remains the best genetic engineering tool available for the duration of the patent, then whoever owns the intellectual rights to CRISPR based therapeutics will control the next 20 years of gene editing therapeutics. Such therapeutics—single treatment cures to genetic based diseases-- could immensely improve human health.However, it is worth remembering that Transcription Activator Like Endonucleases (TALENs) and Zinc-Finger Nucleases (ZFNs) were each once the best available tool, now seen as old news compared with CRISPR. It is plausible that the awareness and value generated by CRISPR/Cas9 will spur greater interest in and funding of molecular biology research, resulting in the production of new discoveries and intellectual property that devalues CRISPR Cas systems. Alternatively, modifications to the CRISPR/Cas9 system may differentiate it sufficiently to create new intellectual property.In the SEC filing, Intellia described its general intended use of funds from the IPO as to advance current candidate products through R&D, submit one or more INDs (Investigational New Drug Application), develop additional in-vivo and ex-vivo product candidates, and further develop its CRISPR/Cas9 gene editing technology.Intellia indicates that its future profitability, among other factors, depends largely on further developing its CRISPR/Cas9 technology, negotiating collaborations favorably, selecting and investing in the right product candidates, advancing them through clinical trials and regulatory approval followed by commercializing and scaling, and protecting and expanding its intellectual property portfolio.Intellia’s process for selecting the best target therapies is to evaluate the type of edit-knockout, repair or insertion, the delivery modality, the presence of established therapeutic endpoints, and the potential for a CRISPR/Cas9 therapy to offer greater benefit than existing technology like TALENs or ZFNs.The company’s five-year collaboration with Novartis began in December of 2014 to develop ex vivo therapies using CRISPR/Cas9 to edit chimeric antigen receptor T cells (CAR T-cells) and Hematopoietic stem cells (HSCs). Intellia is entitled to receive $20 million in technology access fees, $20 million in research payments, and can earn development, regulatory, and sales milestone payments.Just this month, Intellia entered a licensing deal and collaboration with Regeneron to work on gene edits in the liver as well as joint research and development to improve the CRISPR/Cas system. Intellia stands to receive a $75 million payment and, on a per-license basis, can earn payments for development, regulatory, and sales-based milestones.Beyond the work with Novartis and Regeneron, Intellia’s proprietary work includes alpha-1 antitrypsin deficiency, hepatitis B virus, and inborn errors of metabolism.In selecting product candidates, advancing the proprietary and collaborative components of its pipeline, managing the costs of future scaling and commercialization, and protecting its intellectual property, Intellia faces substantial long term challenges with great potential reward.

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